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Item 7. Management’s Discussion and Analysis

2025-03-04 compared with 2024-03-06 · 363 added, 253 removed, 1281 unchanged (32% of the section changed)

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As a result of many factors, including those factors set forth in the “Risk Factors” section of this Annual Report on Form 10-K, our actual results could differ materially from the results described in, or implied by, these forward-looking statements.

We are a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines (also known as oligonucleotides), or those targeting ribonucleic acid (“RNA”), to transform human health.

Our RNA medicines platform, PRISM TM , combines multiple modalities, chemistry innovation and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders.

Our toolkit of RNA-targeting modalities includes RNA editing, splicing, antisense silencing and RNA interference (“RNAi"), providing us with unique capabilities for designing and sustainably delivering candidates that optimally address disease biology.

Our lead programs are in rare and prevalent diseases, including alpha-1 antitrypsin deficiency (“AATD”), obesity, Duchenne muscular dystrophy (“DMD”), and Huntington’s disease (“HD”).

We are a clinical-stage biotechnology company focused on unlocking the broad potential of ribonucleic acid (“RNA”) medicines (also known as oligonucleotides), or those targeting RNA, to transform human health.

Our RNA medicines platform, PRISM ® , combines multiple modalities, chemistry innovation and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and common disorders.

Our toolkit of RNA-targeting modalities includes RNA editing, splicing, silencing using RNA interference (“siRNA") and antisense silencing, providing us with unique capabilities for designing and sustainably delivering candidates that optimally address disease biology.

Our diversified pipeline includes clinical programs in obesity, alpha-1 antitrypsin deficiency (“AATD”), Duchenne muscular dystrophy (“DMD”), and Huntington’s disease (“HD”), as well as several preclinical programs utilizing our versatile RNA medicines platform.

We were founded on the recognition that there was a significant, untapped opportunity to use chemistry innovation to tune the pharmacological properties of oligonucleotides.

Today, we have more than a decade of experience challenging convention related to oligonucleotide design and pioneering novel chemistry modifications to optimize the pharmacological properties of our molecules.

We have seen preclinically and in clinical trials that these chemistry modifications enhance potency, distribution, and durability of effect of our molecules.

Our novel chemistry also allows us to avoid using complex delivery vehicles, such as lipid nanoparticles and viruses, and instead use clinically proven conjugates (e.g.

N -acetylgalactosamine or (“GalNAc”)) or free uptake for delivery to a variety of cell and tissue types.

We have more than a decade of experience challenging convention related to oligonucleotide design and pioneering novel chemistry modifications to optimize the pharmacological properties of our molecules.

We have seen in clinical trials that these chemistry modifications enhance potency, distribution, and durability of effect of our molecules.

Our novel chemistry also allows us to avoid using complex delivery vehicles, such as lipid nanoparticles and viruses, and instead use clinically proven conjugates ( e.g., N -acetylgalactosamine or (“GalNAc”)) or free uptake for delivery to a variety of cell and tissue types.

We maintain strong and broad intellectual property, including for our novel chemistry modifications.

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By opening up new areas of biology, we have also opened up new opportunities to slow, stop or reverse disease and have expanded the possibilities offered through our platform.

The inspiration for our multimodal platform is based on the recognition that the biological machinery (i.e.

enzymes) needed to address human disease already exists within our cells and can be harnessed for therapeutic purposes with the right tools.

The inspiration for our multimodal platform is based on the recognition that the biological machinery (i.e., enzymes) needed to address human disease already exists within our cells and can be harnessed for therapeutic purposes with the right tools.

We believe that we have built the most versatile toolkit of RNA-targeting modalities in the industry, with multiple means of repairing, restoring, or reducing proteins and designing best-fit solutions based on the unique biology of a given disease target.

We are actively advancing programs in all of our modalities.

We are actively advancing programs using four distinct modalities, including novel A-to-I RNA editing oligonucleotides (“AIMers”).

These modalities include:

• RNA editing, which uses AIMers that are designed to target single bases on an RNA transcript and recruit endogenous ADAR enzymes that naturally possess the ability to change an adenine (A) to an inosine (I), which cells read as guanine (G).

This approach enables both the correction of G-to-A point mutations and the modulation of RNA to either upregulate protein expression, modify protein-protein interactions, or alter RNA folding and processing.

AIMers are short in length, fully chemically modified, and use our novel chemistry, which make them distinct from other ADAR-mediated editing approaches.

• Antisense (silencing) , which uses our oligonucleotide designed to bind to a specific sequence in a target RNA strand that encodes a disease-associated protein or pathogenic RNA.

The resulting double-stranded molecule (“duplex”) is then recognized by a cellular enzyme called RNase H, which cleaves, or cuts, the target RNA in the duplex, thereby preventing the disease-associated protein from being made.

• RNA interference (RNAi ) (silencing) , which uses our double-stranded RNAs called siRNAs to engage the RNAi machinery known as the RNA-induced silencing complex (“RISC”) and to silence a target RNA that is either pathogenic itself or encodes a disease-associated protein, thereby preventing the accumulation of the pathogenic species (RNA or protein).

• Splicing / exon skipping , which is the processing of a nascent pre-mRNA transcript into mRNA by removing introns and joining exons together.

Exon skipping uses our oligonucleotide designed to bind to a particular sequence within a target pre-mRNA and direct the cellular machinery to alter the final composition of exons in mature mRNA by deleting, or splicing out, certain specific regions of that RNA.

We intentionally focus on targeting the transcriptome using oligonucleotides rather than other nucleic acid modalities such as gene therapy and DNA editing.

This focus enables us to:

• Leverage diversity of expression across cell types by modulating the many regulatory pathways that impact gene expression, including transcription, endogenous RNA interference pathways, splicing, and translation;

• Leverage diversity of expression across cell types by modulating the many regulatory pathways that impact gene expression, including transcription, endogenous RNAi pathways, splicing, and translation;

• Address diseases that have historically been difficult to treat with small molecules or biologics;

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• Leverage well-established industry manufacturing processes and regulatory, access, and reimbursement pathways.

We have a robust and diverse pipeline of potential first-or best-in-class programs, including:

We have a robust and diverse pipeline of potential first-or best-in-class programs addressing both rare and common diseases:

• GalNAc-conjugated oligonucleotides for hepatic and metabolic diseases including:

WVE-006 is a GalNAc-conjugated SERPINA1 RNA editing oligonucleotide.

Lead clinical candidate is a GalNAc-conjugated RNAi oligonucleotide targeting inhibin βE (“INHBE”).

WVE-007 is a GalNAc-conjugated siRNA targeting inhibin βE (“INHBE”);

• Alpha-1 antitrypsin deficiency ("AATD"):

WVE-006 is a GalNAc-conjugated SERPINA1 AIMer;

• Liver disease:

GalNAc-conjugated AIMer targeting PNPLA3 I148M for correction;

• Heterozygous Familial Hypercholesterolemia (“HeFH”):

GalNAc-conjugated AIMer targeting low-density lipoprotein receptor (“LDLR”) for upregulation and GalNAc-conjugated AIMer targeting apolipoprotein B (“APOB”) for correction.

• Unconjugated oligonucleotides for muscle, CNS and other disease areas including:

• Duchenne muscular dystrophy ("DMD"):

WVE-N531 is an exon 53 splicing oligonucleotide;

WVE-003 is a selective mutant huntingtin (“mHTT”)-lowering SNP3 antisense silencing oligonucleotide.

We are also building a pipeline of novel A-to-I RNA editing oligonucleotides (“AIMers”).

Our RNA editing capability affords us the dexterity to address both rare diseases, as well as those diseases impacting large patient populations.

• Huntington’s disease ("HD"):

WVE-003 is an allele-selective oligonucleotide designed to lower mutant huntingtin (“mHTT”) protein and preserve healthy, wild-type huntingtin (“wtHTT”) protein.

Our RNA editing capability affords us the dexterity to address both rare and common diseases, as well as those diseases impacting large patient populations.

AIMers are designed to target single bases on an RNA transcript and recruit proteins that exist in the body, called ADAR enzymes, which naturally possess the ability to change an adenine (A) to an inosine (I), which cells read as guanine (G).

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AIMers are short in length, fully chemically modified, and use our novel chemistry, which make them distinct from other ADAR-mediated editing approaches.

GSK Collaboration

In December 2022, we announced a strategic collaboration with GlaxoSmithKline Intellectual Property (No.

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Our GSK collaboration has three components:

(1) A discovery collaboration which enables us to advance up to three programs leveraging targets informed by GSK’s novel insights, the first of which is our INHBE program for obesity and other metabolic disorders;

(1) a discovery collaboration which enables us to advance up to three programs leveraging targets informed by GSK’s novel genetic insights;

(2) a discovery collaboration which enables GSK to advance up to eight programs leveraging PRISM and our oligonucleotide expertise and discovery capabilities;

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We will maintain development responsibilities for WVE-006 through completion of RestorAATion-2, at which point development and commercial responsibilities will transition to GSK.

Recent Developments

As previously disclosed, on December 11, 2023, we closed an underwritten public offering (the “December 2023 Offering”) of 20,000,000 of our ordinary shares at a price to the public of $5.00 per ordinary share for gross proceeds of $100.0 million.

2024, the representatives of the underwriters in the December 2023 Offering exercised their option to purchase an additional 3,000,000 ordinary shares, for additional gross proceeds of $15.0 million.

Takeda Collaboration (expired in October 2024)

In February 2018, we entered into a global strategic collaboration with Takeda Pharmaceutical Company Limited (“Takeda”), pursuant to which we agreed to collaborate with Takeda on the research, development and commercialization of oligonucleotide therapeutics for disorders of the CNS.

On October 11, 2024, we were notified by Takeda that Takeda did not intend to exercise and therefore elected to terminate its option for the HD target under the collaboration.

As HD was the last active collaboration target under the collaboration, the collaboration expired with immediate effect.

As a result of the option termination, we are now free to advance WVE-003, our clinical-stage Huntington’s disease program, as well as any other programs targeting HTT, independently or with other partners.

Financial Operations Overview

We have never been profitable, and since our inception, we have incurred significant operating losses.

Our net loss was $57.5 million in 2023 and $161.8 million in 2022.

Our net loss was $97.0 million in 2024, $57.5 million in 2023, and $161.8 million in 2022.

As of December 31, 2024 and 2023, we had an accumulated deficit of $1,121.9 million and $1,024.9 million, respectively.

We expect to incur significant expenses and operating losses for the foreseeable future.

We recognize collaboration revenue under the GSK Collaboration Agreement, which became effective in January 2023, and the Takeda Collaboration Agreement, which became effective in April 2018, (both of which are defined in Note 5 in the notes to the consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K).

We recognize collaboration revenue under the GSK Collaboration Agreement, which became effective in January 2023, and the Takeda Collaboration Agreement, which became effective in April 2018 and expired in the fourth quarter of 2024, (both of which are defined in Note 5 in the notes to the consolidated financial statements appearing elsewhere in this Annual Report on Form 10-K).

We have not generated any product revenue since our inception and do not expect to generate any revenue from the sale of products for the foreseeable future.

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Our primary research and development focus has been the development of our RNA medicines platform, PRISM.

We are using PRISM, which includes our novel chemistry modifications, to design, develop and commercialize a broad pipeline of first- or best- in class RNA medicines using our editing, RNAi, splicing, and antisense modalities.

We are using PRISM, which includes our novel chemistry modifications, to design, develop and commercialize a broad pipeline of first- or best-in class RNA medicines using our editing, splicing, RNAi, and antisense modalities.

Our research and development expenses consist primarily of expenses related to our CROs, CMOs, consultants, other external vendors and fees paid to global regulatory agencies to conduct our clinical trials, in addition to compensation-related expenses, internal manufacturing expenses, facility-related expenses and other general operating expenses.

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However, we do not allocate compensation-related expenses, internal manufacturing expenses, equipment repairs and maintenance expense, facility-related expenses or other operating expenses to specific programs.

These expenses, which are not allocated on a program-by-program basis, are included in the “Other research and development expenses (1) , including INHBE, RNA editing, PRISM, others” category along with other external expenses related to our discovery and development programs, as well as platform development and identification of potential drug discovery candidates.

These expenses, which are not allocated on a program-by-program basis, are included in the “Other research and development expenses (1) , including INHBE, RNA

editing, PRISM, others” category along with other external expenses related to our discovery and development programs, as well as platform development and identification of potential drug discovery candidates.

Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials.

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General and Administrative Expenses

General and administrative expenses consist primarily of compensation-related expenses, including salaries, bonuses, share-based compensation and other related benefits costs for personnel in our executive, finance, corporate, legal and administrative functions, as well as compensation-related expenses for our board of directors.

General and administrative expenses also include legal fees;

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Results of Operations

In this section, we discuss the results of our operations for the year ended December 31, 2023 compared to the year ended December 31, 2022 .

In this section, we discuss the results of our operations for the year ended December 31, 2024 compared to the year ended December 31, 2023 and for the year ended December 31, 2023 compared to the year ended December 31, 2022 .

Comparison of the Year Ended December 31, 2024 to the Year Ended December 31, 2023

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Income tax benefit (provision)

Revenue for the years ended December 31, 2024 and 2023, was $108.3 million and $113.3 million, respectively, and was earned under the GSK Collaboration Agreement and the Takeda Collaboration Agreement.

The $5.0 million decrease in revenue year over year was driven by the revenue recognized under the GSK Collaboration Agreement, partially offset by the increase in revenue recognized under the Takeda Collaboration Agreement.

The $108.3 million in revenue recognized during the year ended December 31, 2024 was comprised of $37.0 million in revenue recognized under the GSK Collaboration Agreement and $71.3 million of revenue recognized under the Takeda Collaboration Agreement.

The $113.3 million in revenue recognized during the year ended December 31, 2023 was comprised of $66.3 million in revenue recognized under the GSK Collaboration Agreement and $47.0 million of revenue recognized under the Takeda Collaboration Agreement.

The increase in the Takeda Collaboration revenue earned year over year was primarily due to the termination of the collaboration in October 2024, which led to the recognition of the remainder of the deferred revenue related to the research and development services, as well as the license related to the HD program.

This was offset by the decrease in the GSK revenue earned year over year primarily related to the AATD program.

Research and Development Expenses

The following table summarizes our research and development expenses incurred for the years ended December 31, 2024 and 2023:

For the Year Ended December 31,

(in thousands)

Other research and development expenses (1) , including INHBE, RNA editing, PRISM, others

ALS and FTD programs (discontinued)

Total research and development expenses

(1) Includes expenses related to other research and development programs, identification of potential drug discovery candidates, compensation-related expenses, internal manufacturing expenses, equipment repairs and maintenance expense, facility-related expenses, and other operating expenses, which are not allocated to specific programs.

Research and development expenses were $159.7 million for the year ended December 31, 2024, compared to $130.0 million for the year ended December 31, 2023.

The increase of $29.7 million was due to the following:

• an increase of $3.2 million in external expenses related to our AATD program, WVE-006 (RNA editing);

• an increase of $7.7 million in external expenses related to our DMD programs, including WVE-N531 (splicing);

• a decrease of $1.3 million in external expenses related to our HD programs, including WVE-003 (silencing);

• an increase of $28.4 million in other research and development expenses, including INHBE, RNA editing, PRISM, and other internal and external research and development expenses that are not allocated on a program-by-program basis or are related to other discovery and development programs, and the identification of potential drug discovery candidates, mainly due to increases in compensation-related expenses and facilities-related expenses, partially offset by decreases in other external research and development expenses;

• a decrease of $8.3 million in external expenses related to our discontinued ALS and FTD program, WVE-004.

General and Administrative Expenses

General and administrative expenses were $59.0 million for the year ended December 31, 2024, compared to $51.3 million for the year ended December 31, 2023.

The increase of $7.7 million is primarily driven by increases in compensation related expenses and administrative expenses.

Other Income, Net

Other income, net for the years ended December 31, 2024 and 2023 was $13.4 million and $9.8 million, respectively.

The increase of $3.6 million in other income, net was primarily driven by an increase in estimated refundable tax credits as well as an increase in dividend income during the year ended December 31, 2024.

Income Tax Benefit

During the years ended December 31, 2024 and 2023, we recorded no income tax benefit or provision and an income tax benefit of $0.7 million, respectively.

The income tax benefit for the year ended December 31, 2023 was due to a change in estimate in connection with U.S.

tax guidance relating to the capitalization of research and development expenditures.

Comparison of the Year Ended December 31, 2023 to the Year Ended December 31, 2022

The following table summarizes our results of operations for 2023 and 2022:

For the Year Ended December 31,

(in thousands)

Operating expenses:

Research and development

General and administrative

Total operating expenses

Loss from operations

Total other income, net

Loss before income taxes

Income tax benefit (provision)

Revenue for the year ended December 31, 2023 was $113.3 million and was earned under the GSK Collaboration Agreement and the Takeda Collaboration Agreement.

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Liquidity and Capital Resources

Since our inception, we have not generated any product revenue and have incurred recurring net losses.

Since our inception, we have not generated any product revenue and have incurred recurring net operating losses.

To date, we have primarily funded our operations through public and other registered offerings of our ordinary shares and other securities, collaborations with third parties and private placements of debt and equity securities.

Through December 31, 2024, we have received an aggregate of approximately $1,578.4 million in net proceeds from these transactions, consisting of $977.8 million in net proceeds from public and other registered offerings of our ordinary shares and other securities, $511.3 million from our collaborations and $89.3 million in net proceeds from private placements of our debt and equity securities.

In January 2024, the representatives of the underwriters in connection with the previously disclosed underwritten public offering (the “December 2023 Offering”) exercised their option to purchase an additional 3,000,000 ordinary shares at a price of $5.00 per ordinary share as a part of the December 2023 Offering.

We received an additional $14.0 million in net proceeds from the December 2023 Offering in January 2024.

On September 27, 2024, we closed the September 2024 Offering in which we issued and sold 23,125,001 of our ordinary shares and the 2024 Pre-Funded Warrants to purchase up to 1,875,023 of our ordinary shares.

The gross proceeds to us from the September 2024 Offering were $200.0 million before deducting underwriting discounts and commissions and other offering expenses.

On October 1, 2024, the representatives of the underwriters exercised their option in full to purchase an additional 3,750,000 ordinary shares, for additional net proceeds to us of approximately $28.2 million.

As of December 31, 2024, we had cash and cash equivalents of $302.1 million, restricted cash of $3.8 million and an accumulated deficit of $1,121.9 million.

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We have based this expectation on assumptions that may prove to be incorrect, and we may use our available capital resources sooner than we currently expect.

In addition, we may elect to raise additional funds before we need them if the conditions for raising capital are favorable due to market conditions or strategic considerations, even if we expect we have sufficient funds for our current or future operating plans.

In addition, we may elect to raise additional funds before we need them if the conditions for raising capital are

favorable due to market conditions or strategic considerations, even if we expect we have sufficient funds for our current or future operating plans.

Our operating lease commitments as of December 31, 2024 total $29.1 million, of which $9.6 million is related to payments in 2025 and approximately $19.5 million is related to payments beyond 2025.

Until we can generate significant revenue from product sales, if ever, we expect to continue to finance our operations through a combination of public or private equity or debt financings or other sources, which may include collaborations with third parties.

In May 2019, we filed a shelf registration statement on Form S-3ASR with the SEC pursuant to which we registered for sale an indeterminate amount of any combination of our ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that we may determine.

Our shelf registration statement on Form S-3ASR also included a prospectus covering up to an aggregate of $250.0 million in ordinary shares that we could issue and sell from time to time, through Jefferies LLC (“Jefferies”) acting as our sales agent, pursuant to the open market sales agreement that we entered into with Jefferies in May 2019, as amended in March 2020 and March 2022 (the “Sales Agreement”), for our “at-the-market” equity program.

Since we no longer qualified as a “well-known seasoned issuer” at the time of the filing of our Annual Report on Form 10-K for the year ended December 31, 2019, we previously amended the shelf registration statement to register for sale up to $500.0 million of any combination of our ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that we may determine, including the $250.0 million in ordinary shares that we may issue and sell from time to time pursuant to our “at-the-market” equity program.

This registration statement, which we refer to as the “2019 Form S-3,” remained effective until our 2022 Form S-3 (as defined below) was declared effective on May 4, 2022, after which time we may no longer offer or sell any securities under the 2019 Form S-3.

On March 3, 2022, we filed a new universal shelf registration on Form S-3 with the SEC, which was declared effective by the SEC on May 4, 2022, pursuant to which we registered for sale up to $500.0 million of any combination of our ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that we may determine, which we refer to as the “2022 Form S-3.” The 2022 Form S-3 includes a prospectus covering up to approximately $132.0 million in ordinary shares that had not yet been issued or sold under our Sales Agreement with Jefferies at the time the 2022 Form S-3 was declared effective.

During the year ended December 31, 2023, we sold 751,688 ordinary shares under our at-the-market equity program for aggregate gross proceeds of $3.3 million.

As of March 1, 2024 we have $311.7 million in securities available for issuance under the 2022 Form S-3, including approximately $128.7 million in ordinary shares available for issuance under our at-the-market equity program.

On November 12, 2024, we filed a shelf registration statement on Form S-3ASR with the SEC for which we registered for sale an indeterminate amount of any combination of our ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that we may determine, which we refer to as the “2024 WKSI Shelf”.

Our 2024 WKSI Shelf includes a prospectus covering up to an aggregate of $250.0 million in ordinary shares that we are able to issue and sell from time to time, through Jefferies LLC (“Jefferies”) acting as our sales agent, pursuant to the Open Market Sale Agreement, dated May 10, 2019, as amended by Amendment No.

1, dated as of March 2, 2020, Amendment No.

2, dated as of March 3, 2022, and Amendment No.

3, dated November 12, 2024, (collectively, the “Sales Agreement”), for our “at-the-market” equity program.

For the three months ended December 31, 2024, we received $5.2 million in net proceeds from sales under our “at-the-market" equity program.

Adequate additional financing may not be available to us on acceptable terms, or at all.

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The non-cash charges for 2024 related to share-based compensation expense of $13.1 million, amortization of right-of-use assets of $4.8 million, and depreciation expense of $3.9 million.

The largest change in operating assets and liabilities was a $93.6 million decrease in deferred revenue, mainly driven by our Takeda Collaboration Agreement, which was partially offset by the second largest change in operating assets and liabilities, the $19.7 million decrease in accounts receivable primarily due to the collection of receivables related to the GSK Collaboration Agreement.

During 2023, operating activities used $19.4 million of cash, primarily due to our net loss of $57.5 million, partially offset by non-cash charges of $19.0 million and changes in our operating assets and liabilities of $19.1 million.

The non-cash charges for 2023 related to share-based compensation expense of $9.8 million, amortization of right-of-use assets of $4.2 million, and depreciation expense of $5.0 million.

The largest change in operating assets and liabilities was a $54.3 million increase in deferred revenue, mainly driven by our GSK Collaboration Agreement, which became effective in January 2023, which was partially offset by the second largest change in operating assets and liabilities, the $21.1 million increase in accounts receivable primarily related to the achievement of a milestone under the GSK Collaboration Agreement.

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During 2023, investing activities used $1.1 million of cash, primarily consisting of purchases of property and equipment.

During 2022, investing activities used $1.3 million of cash, primarily consisting of purchases of property and equipment.

Additionally, we purchased $75.0 million of short-term investments during 2022, all of which matured in 2022.

Financing Activities

During 2024, net cash provided by financing activities was $253.9 million, which was primarily due to the $215.8 million in net proceeds from the September 2024 Offering of ordinary shares and the 2024 Pre-Funded Warrants;

as well as the $14.0 million in net proceeds from the January 2024 exercise of the underwriters’ option to purchase an additional 3,000,000 shares under the December 2023 Offering.

Additionally, we received $20.4 million in net proceeds from sales under our “at-the-market” equity program.

During 2023, net cash provided by financing activities was $132.5 million, primarily due to the $93.6 million in net proceeds from the December 2023 Offering, which comprised of sales of ordinary shares, as well as $34.6 million in net proceeds from the GSK Equity Investment.

Additionally, there were $3.1 million in net proceeds from our "at-the-market" equity program.

During 2022, net cash provided by financing activities was $67.2 million, primarily due to the $65.5 million in net proceeds from the underwritten offering we completed in June 2022, which was comprised of sales of ordinary shares and pre-funded warrants.

Additionally, there were $1.1 million in net proceeds from our "at-the-market" equity program.

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• seek and obtain regulatory approvals for our product candidates;

• respond to the impacts of local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, rising inflation, rising interest rates or market disruptions on our business;

• respond to the impacts of local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, volatility in inflation, volatility in interest rates or market disruptions on our business;

• establish and build capabilities to market, distribute and sell our product candidates.

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• our ability to obtain marketing approval for our product candidates;

• the impacts of local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, rising inflation, rising interest rates or market disruptions on our business;

• the impacts of local and global health epidemics, the conflict involving Russia and Ukraine, the conflict in the Middle East, global economic uncertainty, volatility in inflation, volatility in interest rates or market disruptions on our business;

• the costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval;

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Adequate additional funds may not be available to us on acceptable terms when we need them, or at all.

We do not currently have any committed external source of funds, except for possible future payments from Takeda or GSK under our collaborations with them.

We do not currently have any committed external source of funds, except for possible future payments from GSK under our collaborations with them.

To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing shareholders will be diluted, and the terms may include liquidation or other preferences that adversely affect the rights of our shareholders.

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Critical Accounting Policies and Significant Judgments and Estimates

Our consolidated financial statements are prepared in accordance with generally accepted accounting principles in the United States of America (“U.S.

Our consolidated financial statements are prepared in accordance with U.S.

The preparation of our financial statements and related disclosures requires us to make estimates and assumptions that affect the reported amount of assets, liabilities, revenue, costs and expenses and related disclosures.

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(a) the number of performance obligations based on the determination under step (ii) above;

(b) the transaction

price under step (iii) above;

(b) the transaction price under step (iii) above;

and (c) the timing of satisfaction of performance obligations as a measure of progress in step (v) above.

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For research and development services that are combined with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue.

The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition.

Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition.

Customer options:

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Our cash and cash equivalents are comprised of funds held in checking accounts and money market accounts.

Our short-term investments were comprised of term deposits which had fixed interest rates.

There were no short-term investments as of December 31, 2023 or 2022, as the $75.0 million of term deposits that constitute the Company’s short-term investments were purchased and all matured during the twelve months ended December 31, 2022.

Foreign Currency Risk

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For the years ended December 31, 2024, 2023, and 2022, changes in foreign currency exchange rates did not have a material impact on our historical financial position, our business, our financial condition, our results of operations or our cash flows.

A hypothetical 10% change in foreign currency rates would not have a material impact on our historical financial position or results of operations.

However, there can be no assurance that changes in foreign currency exchange rates will not have a material adverse impact on us in the future.

Inflation Risk

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Our management is responsible for establishing and maintaining adequate internal control over financial reporting.

Internal control over financial reporting is defined in Rules 13a-15(f) and 15d-15(f) under the Securities Exchange Act of 1934, as amended, as a process designed by, or under the supervision of, the company’s principal executive and principal financial officers and effected by the company’s board of directors, management and other personnel to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles and includes those policies and procedures that:

Internal control over financial reporting is defined in Rules 13a-15(f) and 15d-15(f) under the Securities Exchange Act of 1934, as amended, as a process designed by, or under the supervision of, the company’s principal executive and principal financial officers and effected by the company’s Board, management and other personnel to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles and includes those policies and procedures that:

• Pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company;

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Based on our assessment, management believes that, as of December 31, 2024, our internal control over financial reporting is effective based on those criteria.

As a “non-accelerated filer,” we are exempt from the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act of 2002.

As a result, KPMG LLP, our independent registered public accounting firm, has not audited or issued an attestation report with respect to the effectiveness of our internal control over financial reporting as of December 31, 2023.

The effectiveness of our internal control over financial reporting as of December 31, 2024 has been audited by KPMG LLP, an independent registered public accounting firm, as stated in their report.

Other Information

Singapore Goods and Services Tax (“GST”) Rate

The Singapore GST rate was increased from 8% to 9% from January 1, 2024.

The issue or transfer of ownership of our ordinary shares would be exempt from GST, although the sale of our ordinary shares by a GST-registered investor may be considered to be a taxable supply subject to GST at 0% if certain conditions are met.

Services consisting of arranging, brokering, underwriting or advising on the issue, allotment or transfer of ownership of our ordinary shares rendered by a GST-registered person to an investor belonging in Singapore for GST purposes in connection with the investor’s purchase, sale or holding of our ordinary shares will be subject to GST at the standard rate.

Similar services rendered by a GST-registered person contractually to an investor belonging outside Singapore and for the direct benefit of an investor belonging outside Singapore or a GST-registered person in Singapore should generally, subject to the satisfaction of certain conditions, be subject to GST at 0%.

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We describe the material terms of these Rule 10b5-1 Trading Plans below.

On November 21, 2023 , Paul B.

Bolno , M.D., MBA, our President and Chief Executive Officer , adopted a Rule 10b5-1 Trading Plan providing for the sale of up to an aggregate of 435,594 of our ordinary shares pursuant to the terms of such Rule 10b5-1 Trading Plan.

Bolno's Rule 10b5-1 Trading Plan is active until June 25, 2024 , or earlier, if and when all transactions under the Rule 10b5-1 Trading Plan are completed.

On November 21, 2023 , Christopher Francis , Ph.D., our Senior Vice President , Corporate Development, Head of Emerging Areas, adopted a Rule 10b5-1 Trading Plan providing for the sale of up to an aggregate of 244,836 of our ordinary shares pursuant to the terms of such Rule 10b5-1 Trading Plan.

Francis' Rule 10b5-1 Trading Plan is active until February 28, 2025 , or earlier, if and when all transactions under the Rule 10b5-1 Trading Plan are completed.

On November 24, 2024 , Christian Henry , MBA, Chairman of our Board , adopted a Rule 10b5-1 Trading Plan providing for the sale of up to an aggregate of 105,670 of our ordinary shares pursuant to the terms of such Rule 10b5-1 Trading Plan.

10b5-1 Trading Plan is active until August 29, 2025 , or earlier, if and when all transactions under the Rule 10b5-1 Trading Plan are completed.

On November 25, 2024 , Kyle Moran , CFA, our Chief Financial Officer , adopted a Rule 10b5-1 Trading Plan providing for the sale of up to an aggregate of 196,000 of our ordinary shares pursuant to the terms of such Rule 10b5-1 Trading Plan.

Moran’s Rule 10b5-1 Trading Plan is active until October 31, 2024 , or earlier, if and when all transactions under the Rule 10b5-1 Trading Plan are completed.

Moran’s Rule 10b5-1 Trading Plan is active until June 30, 2025 , or earlier, if and when all transactions under the Rule 10b5-1 Trading Plan are completed.

On November 25, 2024 , Christopher Francis , Ph.D., our Senior Vice President , Corporate Development, Head of Emerging Areas, adopted a Rule 10b5-1 Trading Plan providing for the sale of up to an aggregate of 435,564 of our ordinary shares pursuant to the terms of such Rule 10b5-1 Trading Plan.

Francis' Rule 10b5-1 Trading Plan is active until November 3, 2025 , or earlier, if and when all transactions under the Rule 10b5-1 Trading Plan are completed.

On November 25, 2024 , Chandra Vargeese , Ph.D., our Chief Technology Officer , Head of Platform Discovery Sciences, adopted a Rule 10b5-1 Trading Plan providing for the sale of up to an aggregate of 86,972 of our ordinary shares pursuant to the terms of such Rule 10b5-1 Trading Plan.

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Directors, Executive Off icers and Corporate Governance

Information required by this item will be contained in our definitive proxy statement to be filed with the SEC on Schedule 14A in connection with our 2024 Annual General Meeting of Shareholders, or the Proxy Statement, if the Proxy Statement is filed not later than 120 days after the end of our fiscal year ended December 31, 2023, in the sections titled “Management and Corporate Governance,” and “Code of Business Conduct and Ethics,” and is incorporated herein by reference.

If the Proxy Statement is not filed within such 120-day period, the information required by this item will be contained in an amendment to this Annual Report on Form 10-K to be filed with the SEC, or the Form 10-K/A.

Information required by this item will be contained in our definitive proxy statement to be filed with the SEC on Schedule 14A in connection with our 2025 Annual General Meeting of Shareholders, (the "Proxy Statement"), if the Proxy Statement is filed not later than 120 days after the end of our fiscal year ended December 31, 2024, in the sections titled “Management and Corporate Governance,” and “Code of Business Conduct and Ethics,” and is incorporated herein by reference.

Executi ve Compensation

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(Exhibit 4.1)

Investors’ Rights Agreement by and among the Registrant and certain of its shareholders, dated as of August 14, 2015

(Exhibit 4.2)

Amendment No.

1 to Investors’ Rights Agreement by and among the Registrant and certain of its shareholders, dated as of November 8, 2018

Form of Pre-Funded Warrant (2024)

(Exhibit 4.1)

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(Exhibit 10.5)

Collaboration and License Agreement by and between Wave Life Sciences USA, Inc., Wave Life Sciences UK Limited and Takeda Pharmaceutical Company Limited, dated as of February 19, 2018

(Exhibit 10.1)

First Amendment to Collaboration and License Agreement by and between Wave Life Sciences USA, Inc., Wave Life Sciences UK Limited and Takeda Pharmaceutical Company Limited, dated as of August 4, 2020

(Exhibit 10.3)

Second Amendment to Collaboration and License Agreement by and between Wave Life Sciences USA, Inc., Wave Life Sciences UK Limited and Takeda Pharmaceutical Company Limited, dated as of October 15, 2021

(Exhibit 10.3.2)

Share Purchase Agreement by and between Takeda Pharmaceutical Company Limited and the Registrant, dated as of February 19, 2018

(Exhibit 10.2)

Investor Agreement by and between Takeda Pharmaceutical Company Limited and the Registrant, dated as of April 2, 2018

(Exhibit 10.3)

Agreements with Executive Officers and Directors

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(Exhibit 10.12)

Employment Agreement between the Registrant and Michael Panzara, M.D.

dated as of July 11, 2016

(Exhibit 10.4)

Employment Agreement, as amended and restated, between the Registrant and Kyle Moran, dated as of January 1, 2021

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(Exhibit 10.16)

Nominee Director Fee Agreement by and between the Registrant and Miura & Associates Management Consultants Pte.

Ltd., dated as of October 23, 2012

(Exhibit 10.17)

Equity and Other Compensation Plans

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Wave Life Sciences Ltd.

2021 Equity Incentive Plan, as amended (the “2021 Equity Plan”)

(Exhibit 10.1)

Wave Life Sciences Ltd.

2021 Equity Incentive Plan, as amended, effective as of August 1, 2023

(Exhibit 10.1)

Wave Life Sciences Ltd.

2019 Employee Share Purchase Plan, effective as of August 15, 2019

2021 Equity Plan, as amended, (the “2021 Equity Plan”) effective August 6, 2024

(Exhibit 10.1)

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(Exhibit 10.6)

Form of Inducement Non-qualified Share Option Agreement

Form of Inducement Non-qualified Share Option Agreement, effective May 2024

(Exhibit 10.1)

Form of Inducement Restricted Share Unit Agreement

Form of Inducement Restricted Share Unit Agreement, effective May 2024

(Exhibit 10.2)

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(Exhibit 10.1)

Amendment No.

3, dated November 12, 2024, to the Open Market Sale Agreement, dated as of May 10, 2019, by and between Wave Life Sciences Ltd.

and Jefferies LLC

(Exhibit 10.1)

Insider Trading Policy

List of Subsidiaries of the Registrant

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Clawback Policy, effective as of October 2, 2023

(Exhibit 97.1)

XBRL Instance Document – T he Instance Document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document

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(*) The certification attached as Exhibit 32 that accompanies this Annual Report on Form 10-K is not deemed filed with the SEC and is not to be incorporated by reference into any filing of Wave Life Sciences Ltd.

under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form 10-K, irrespective of any general incorporation language contained in such filing.

under the Securities Act of 1933, as amended, or the

Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form 10-K, irrespective of any general incorporation language contained in such filing.

(+) Indicates management contract or compensatory plan or arrangement.

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Wave Life Sciences Ltd.:

Opinion on the Consolidated Financial Statements

Opinions on the Consolidated Financial Statements and Internal Control Over Financial Reporting

We have audited the accompanying consolidated balance sheets of Wave Life Sciences Ltd.

and subsidiaries (the Company) as of December 31, 2023 and 2022, the related consolidated statements of operations and comprehensive loss, Series A preferred shares and shareholders’ equity (deficit), and cash flows for the years then ended, and the related notes (collectively, the consolidated financial statements).

In our opinion, the consolidated financial statements present fairly, in all material respects, the financial position of the Company as of December 31, 2023 and 2022, and the results of its operations and its cash flows for the years then ended, in conformity with U.S.

and subsidiaries (the Company) as of December 31, 2024 and 2023, the related consolidated statements of operations and comprehensive loss, Series A preferred shares and shareholders’ equity (deficit), and cash flows for the each of the years in the three-year period ended December 31, 2024, and the related notes (collectively, the consolidated financial statements).

We also have audited the Company’s internal control over financial reporting as of December 31, 2024, based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission.

In our opinion, the consolidated financial statements referred to above present fairly, in all material respects, the financial position of the Company as of December 31, 2024 and 2023, and the results of its operations and its cash flows for each of the years in the three-year period ended December 31, 2024, in conformity with U.S.

generally accepted accounting principles.

Basis for Opinion

These consolidated financial statements are the responsibility of the Company’s management.

Our responsibility is to express an opinion on these consolidated financial statements based on our audits.

Also in our opinion, the Company maintained, in all material respects, effective internal control over financial reporting as of December 31, 2024 based on criteria established in Internal Control – Integrated Framework (2013) issued by the Committee of Sponsoring Organizations of the Treadway Commission.

Basis for Opinions

The Company’s management is responsible for these consolidated financial statements, for maintaining effective internal control over financial reporting, and for its assessment of the effectiveness of internal control over financial reporting, included in the accompanying Management's Annual Report on Internal Controls over Financial Reporting.

Our responsibility is to express an opinion on the Company’s consolidated financial statements and an opinion on the Company’s internal control over financial reporting based on our audits.

We are a public accounting firm registered with the Public Company Accounting Oversight Board (United States) (PCAOB) and are required to be independent with respect to the Company in accordance with the U.S.

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We conducted our audits in accordance with the standards of the PCAOB.

Those standards require that we plan and perform the audit to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error or fraud.

The Company is not required to have, nor were we engaged to perform, an audit of its internal control over financial reporting.

As part of our audits, we are required to obtain an understanding of internal control over financial reporting but not for the purpose of expressing an opinion on the effectiveness of the Company’s internal control over financial reporting.

Accordingly, we express no such opinion.

Our audits included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

Those standards require that we plan and perform the audits to obtain reasonable assurance about whether the consolidated financial statements are free of material misstatement, whether due to error or fraud, and whether effective internal control over financial reporting was maintained in all material respects.

Our audits of the consolidated financial statements included performing procedures to assess the risks of material misstatement of the consolidated financial statements, whether due to error or fraud, and performing procedures that respond to those risks.

Such procedures included examining, on a test basis, evidence regarding the amounts and disclosures in the consolidated financial statements.

Our audits also included evaluating the accounting principles used and significant estimates made by management, as well as evaluating the overall presentation of the consolidated financial statements.

We believe that our audits provide a reasonable basis for our opinion.

Our audit of internal control over financial reporting included obtaining an understanding of internal control over financial reporting, assessing the risk that a material weakness exists, and testing and evaluating the design and operating effectiveness of internal control based on the assessed risk.

Our audits also included performing such other procedures as we considered necessary in the circumstances.

We believe that our audits provide a reasonable basis for our opinions.

Definition and Limitations of Internal Control Over Financial Reporting

A company’s internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles.

A company’s internal control over financial reporting includes those policies and procedures that (1) pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of the assets of the company;

(2) provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance with generally accepted accounting principles, and that receipts and expenditures of the company are being made only in accordance with authorizations of management and directors of the company;

and (3) provide reasonable assurance regarding prevention or timely detection of unauthorized acquisition, use, or disposition of the company’s assets that could have a material effect on the financial statements.

Because of its inherent limitations, internal control over financial reporting may not prevent or detect misstatements.

Also, projections of any evaluation of effectiveness to future periods are subject to the risk that controls may become inadequate because of changes in conditions, or that the degree of compliance with the policies or procedures may deteriorate.

Critical Audit Matter

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Specifically, evaluating the estimate of total costs expected to be incurred in satisfying certain R&D performance obligations required especially challenging auditor judgment.

This involved an assessment of the nature of the work to be performed and the method for measuring progress.

This involved an assessment of the nature of work to be performed and the method for measuring progress.

The following are the primary procedures we performed to address this critical audit matter.

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Total long-term assets

Liabilities, Series A preferred shares and shareholders’ equity (deficit)

Liabilities, Series A preferred shares and shareholders’ equity

Current liabilities:

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Operating lease liability, net of current portion

Other liabilities

Total long-term liabilities

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and outstanding at December 31, 2024 and 2023

Shareholders’ equity (deficit):

Shareholders’ equity:

Ordinary shares, no par value;

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Accumulated deficit

Total shareholders’ equity (deficit)

Total liabilities, Series A preferred shares and shareholders’ equity (deficit)

Total shareholders’ equity

Total liabilities, Series A preferred shares and shareholders’ equity

The accompanying notes are an integral part of the consolidated financial statements.

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Dividend income and interest income, net

Other income (expense), net

Other income, net

Total other income, net

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net of offering costs

Issuance of ordinary shares

pursuant to the at-the-market

equity program, net

Issuance of ordinary shares pursuant to

the at-the-market equity program, net

Issuance of pre-funded warrants,

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net of offering costs

Issuance of ordinary shares, pursuant

to the GSK Collaboration Agreement

Issuance of ordinary shares pursuant to

the at-the-market equity program, net

Share-based compensation

Vesting of RSUs

Option exercises

Issuance of ordinary shares

pursuant to the GSK Collaboration

under the ESPP

Other comprehensive loss

Balance at December 31, 2023

Issuance of ordinary shares,

pursuant to the at-the-market

equity program, net

net of offering costs

Issuance of ordinary shares pursuant to

the at-the-market equity program, net

Issuance of pre-funded warrants,

net of offering costs

Share-based compensation

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Cash flows from operating activities

Adjustments to reconcile net loss to net cash

used in operating activities:

Adjustments to reconcile net loss to net cash provided by (used in) operating activities:

Amortization of right-of-use assets

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Cash flows from financing activities

Proceeds from issuance of ordinary shares, net of offering costs

Proceeds from issuance pre-funded warrants, net of offering costs

Proceeds from the issuance of ordinary shares as a part of the

June 2022 Offering, net of offering costs

Proceeds from the issuance of ordinary shares as a part of the

December 2023 Offering, net of offering costs

Proceeds from the issuance of ordinary shares as a part of the

September 2024 Offering, net of offering costs

Proceeds from issuance pre-funded warrants as a part of the

June 2022 Offering, net of offering costs

Proceeds from issuance pre-funded warrants as a part of the,

September 2024 Offering, net of offering costs

Proceeds from issuance of ordinary shares pursuant to the

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Wave Life Sciences Ltd.

(together with its subsidiaries, “Wave” or the “Company”) is a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines (also known as oligonucleotides), or those targeting ribonucleic acid “RNA”), to transform human health.

Wave’s RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders.

The Company’s toolkit of RNA-targeting modalities includes RNA editing, splicing, antisense silencing and RNA interference (“RNAi”), providing the Company with unique capabilities for designing and sustainably delivering candidates that optimally address disease biology.

The Company’s lead programs are in rare and prevalent diseases, including alpha-1 antitrypsin deficiency (“AATD”), obesity, Duchenne muscular dystrophy (“DMD”), and Huntington’s disease (“HD”).

The Company’s toolkit of RNA-targeting modalities includes RNA editing, splicing, silencing using siRNA and antisense silencing, providing us with unique capabilities for designing and sustainably delivering candidates that optimally address disease biology.

The Company’s diversified pipeline includes clinical programs in obesity, AATD, DMD, and HD, as well as several preclinical programs utilizing our versatile RNA medicines platform.

The Company was incorporated in Singapore on July 23, 2012 and has its principal U.S.

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The Company has based this expectation on the best information available, however the Company may use its available capital resources sooner than it currently expects.

In January 2024 , the representatives of the underwriters in connection with the December 2023 Offering (as defined in Note 6) exercised their option to purchase an additional 3,000,000 ordinary shares as a part of the December 2023 Offering, for additional net proceeds of approximately $ 14.0 million.

If the Company’s anticipated operating results are not achieved in future periods, planned expenditures may need to be further reduced in order to extend the time period over which the then-available resources would be able to fund the Company’s operations.

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These efforts require significant amounts of additional capital, adequate personnel infrastructure and extensive compliance-reporting capabilities.

There can be no assurance that the Company’s research and development efforts will be successful, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government regulatory approval or that any approved products will be

commercially viable.

Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales.

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The Company's cash and cash equivalents are comprised of funds held in checking and money market accounts.

Short Term Investments

The Company’s short term investments consist of term deposits.

Principles of Consolidation

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The Company’s actual results may differ from these estimates under different assumptions and conditions.

The Company manages its operations as a single segment for the purposes of assessing performance and making operating decisions.

The Company’s focus is on developing its proprietary RNA medicines platform, PRISM, to develop and commercialize a broad pipeline RNA medicines in a variety of therapeutic areas.

The Company manages its operations as a single reportable and operating segment for the purposes of assessing performance and making operating decisions.

The Company’s focus is on developing its proprietary RNA medicines platform, PRISM, to develop and commercialize a broad pipeline of RNA medicines in a variety of therapeutic areas.

This operating structure enables the Chief Executive Officer ("CEO") as chief operating decision maker ("CODM"), to allocate resources and assess business performance in order to achieve established long-term strategic goals.

The determination of a single segment is consistent with the consolidated financial information regularly reviewed by the CODM for purposes of assessing performance, allocating resources and planning, monitoring budget versus actual results, and forecasting future periods.

Within the single segment, there are significant expenses that are regularly considered by the CODM which are used in the review for performance and resource allocation.

See Note 14 for additional disclosure of our segment information.

Going Concern

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dollar for all of the Company’s entities aside from Wave Japan, which has the Japanese Yen as its functional currency.

Assets and liabilities of Wave Japan are translated at period end exchange rates while revenues and expenses of

Wave Japan are translated at average exchange rates for the period.

Assets and liabilities of Wave Japan are translated at period end exchange rates while revenues and expenses of Wave Japan are translated at average exchange rates for the period.

Net unrealized gains and losses from foreign currency translation are reflected as other comprehensive income (loss) within the consolidated statements of Series A preferred shares and shareholders’ equity (deficit) and the consolidated statements of operations and comprehensive loss.

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Cash, cash equivalents and restricted cash are Level 1 assets which are comprised of funds held in checking and money market accounts.

Short-term investments are Level 2 assets which are comprised of term deposits.

Cash, cash equivalents and restricted cash were recorded at fair value as of December 31, 2024 and 2023, totaling $ 305.8 million and $ 204.1 million, respectively.

There were no short-term investments as of December 31, 2023 or 2022 , as the $ 75.0 million of term deposits that constitute the Company’s short-term investments were all purchased and reached maturity during the twelve months ended December 31, 2022 .

The carrying amounts of accounts payable and accrued expenses approximate their fair values due to their short-term maturities.

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Restricted cash consists primarily of cash placed in separate restricted bank accounts as required under the terms of the Company’s lease agreements for its Cambridge, Massachusetts and Lexington, Massachusetts facilities (refer to Note 8).

As of December 31, 2023 and 2022, the Company had $ 3.7 million of restricted cash, of which $ 2.7 million related to the Lexington facility and $ 1.0 million related to the Cambridge facility.

As of December 31, 2024 and 2023, the Company had $ 3.8 million and $ 3.7 million of restricted cash, respectively, of which $ 2.8 million and $ 2.7 million related to the Lexington facility, respectively, and $ 1.0 million related to the Cambridge facility.

Property and Equipment

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In addition, the Company considers whether the customer can benefit from a promise for its intended purpose without the receipt of the remaining promise, whether the value of the promise is dependent on the unsatisfied promise, whether there are other vendors that could provide the remaining promise, and whether it is separately identifiable from the remaining promise.

For licenses that are combined with other promises, the Company utilizes

judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue.

For licenses that are combined with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue.

The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition.

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Costs associated with licenses of technology and patent costs are expensed as incurred and are generally included in research and development expense in the consolidated statements of operations and comprehensive loss.

Refundable Tax Credits

The Company is eligible for refundable tax credits with tax authorities for certain qualified operating expenses.

The Company recognizes refundable tax credits when there is reasonable assurance that the Company will comply with the requirements of the refundable tax credit and that the refundable tax credit will be received.

Refundable tax credits are recorded as income and classified in other income, net in the consolidated statements of operations and comprehensive loss.

Net Loss per Share

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Diluted net loss per share is computed using the sum of the weighted-average number of ordinary shares outstanding during the period and, if dilutive, the weighted-average number of potential ordinary shares, including the assumed exercise of share options and the assumed vesting of RSUs (as defined in Note 7).

The Company applies the two-class method to calculate its basic and diluted net loss per share attributable to ordinary shareholders, as its Series A preferred shares are participating securities.

The two-class method is an earnings allocation formula that treats a participating security as having rights to earnings that otherwise would have been available to ordinary shareholders.

However, for the periods presented, the two-class method does not impact the net loss per ordinary share as the Company was in a net loss position for each of the periods presented and holders of Series A preferred shares do not participate in losses.

The Company’s Series A preferred shares contractually entitle the holders of such shares to participate in dividends but do not contractually require the holders of such shares to participate in losses of the Company.

Accordingly, for periods in which the Company reports a net loss attributable to ordinary shareholders, diluted net loss per share attributable to ordinary shareholders is the same as basic net loss per share attributable to ordinary shareholders, since dilutive ordinary shares are not assumed to have been issued if their effect is anti-dilutive.

The Company’s Series A preferred shares do not entitle the holders of such shares to participate in dividends and do not contractually require the holders of such shares to participate in losses of the Company.

Share-Based Compensation

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ASU 2023-07 requires enhanced disclosures about reportable segments and the chief operating decision maker.

The new guidance is effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024.

Early adoption is permitted.

The Company is currently evaluating the potential impact that the adoption of ASU 2023-07 may have on its consolidated financial statements.

The Company adopted ASU 2023-07 for the Company's fiscal year 2024 annual reporting period and applied it retrospectively.

The adoption did not have a material impact on the Company's consolidated financial statements.

In December 2023, the FASB finalized Accounting Standards Update No.

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ASU 2023-09 is effective for the Company’s annual reporting periods beginning after December 15, 2025.

Adoption is either with a prospective method or a fully retrospective method of transition.

Adoption is either with a

prospective method or a fully retrospective method of transition.

Early adoption is permitted.

The Company is currently evaluating the effect that adoption of ASU 2023-09 will have on its consolidated financial statements.

In November 2024, the FASB issued ASU 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40) (“ASU 2024-03”).

ASU 2024-03 modifies the rules on income statement disclosures to enhance the transparency of and include more detailed information about the types of expenses, including purchases of inventory, employee compensation, depreciation, amortization, and depletion, in commonly presented expense captions such as cost of sales, research and development, and selling, general and administrative expenses.

The amendments are intended to address investors’ requests for income statement expense disclosures that provide more information to help them better understand the components of an entity’s expenses, make their own judgments about the entity’s performance, and more accurately forecast expenses, and enable investors to better assess an entity’s prospects for future cash flows.

It will also provide contextual information for an entity’s presentation and consideration of management’s discussion and analysis of financial position and results of operations.

The guidance is effective for all entities for annual periods beginning after December 15, 2026.

All entities should apply the guidance prospectively but have the option to apply it retrospectively.

Early adoption is permitted.

The Company is continuing to assess the timing of adoption and the potential impacts of ASU 2024-03 on the consolidated financial statements and related disclosures.

PROPERTY AND EQUIPMENT, NET

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Substantially all of the Company’s long-lived assets were located in the United States as of December 31, 2024 and 2023.

Depreciation expense was $ 5.0 million and $ 6.6 million for the years ended December 31, 2023 and 2022 , respectively.

Depreciation expense was $ 3.9 million, $ 5.0 million, and $ 6.6 million for the years ended December 31, 2024, 2023, and 2022 , respectively.

ACCRUED EXPENSES AND OTHER CURRENT LIABILITIES

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Pursuant to the GSK Collaboration Agreement, Wave and GSK have agreed to collaborate on the research, development, and commercialization of oligonucleotide therapeutics, including an exclusive global license to WVE-006.

The discovery collaboration component has an initial four-year research term and combines Wave’s proprietary discovery and drug development platform, PRISM, with GSK’s unique genetic insights and its global development and commercial capabilities.

development and commercial capabilities.

On January 27, 2023, the GSK Collaboration Agreement became effective, and GSK paid Wave an upfront payment of $ 120.0 million.

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The GSK Equity Investment closed on January 26, 2023, following the completion of customary closing conditions.

The ordinary shares purchased by GGL are subject to lock-up and standstill restrictions and carry certain registration rights, customary for transactions of this kind.

The ordinary shares purchased by GGL in the GSK Equity Investment are subject to lock-up and standstill restrictions and carry certain registration rights, customary for transactions of this kind.

The Company did not incur any material costs in connection with the issuance of the ordinary shares under the SPA.

The GSK Collaboration Agreement has three components:

An exclusive global license for GSK to WVE-006, the Company’s then preclinical, first-in-class A-to-I(G) RNA editing candidate for alpha-1 antitrypsin deficiency (“AATD”), with development and commercialization responsibilities transferring to GSK after the Company completes the first-in-patient study (the “AATD Collaboration”).

(1) a discovery collaboration which enables the Company to advance up to three programs leveraging targets informed by GSK’s novel genetic insights (“Wave’s Collaboration Programs”);

(2) a discovery collaboration which enables GSK to advance up to eight programs leveraging PRISM and the Company’s oligonucleotide expertise and discovery capabilities (the “Discovery Research Collaboration”);

and (3) an exclusive global license for GSK to WVE-006, the Company’s alpha-1 antitrypsin deficiency (“AATD”) program, that uses the Company’s proprietary AIMer technology (the "AATD Collaboration").

The Company will be responsible for preclinical, regulatory, manufacturing, and clinical activities for WVE-006 through the initial Phase 1/2 study, at the Company’s sole cost.

Thereafter, GSK will be responsible for advancing WVE-006 through pivotal studies, registration, and global commercialization at GSK’s sole cost.

A discovery research collaboration which enables GSK to advance up to eight programs leveraging PRISM and the Company’s oligonucleotide expertise and discovery capabilities (the “Discovery Research Collaboration”);

A discovery collaboration which enables the Company to advance up to three programs leveraging targets informed by GSK’s novel genetic insights (“Wave’s Collaboration Programs”).

Under the GSK Collaboration Agreement, each party grants to the other party certain licenses to the collaboration products to enable the other party to perform its obligations and exercise its rights under the GSK Collaboration Agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the GSK Collaboration Agreement.

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Both parties are represented equally for these committees and report to the joint steering committee.

The Company assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, GSK, is a customer for the AATD Collaboration prior to GSK exercising its option and, for the Discovery Research Collaboration programs during the target validation research term.

The Company assessed this arrangement in accordance with ASC 606, Revenue from Contracts with Customers ("ASC 606") and concluded that the contract counterparty, GSK, is a customer for the AATD Collaboration prior to GSK exercising its option and, for the Discovery Research Collaboration programs during the target validation research term.

The Company identified the following material promises under the arrangement:

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At the outset of the arrangement, the transaction price included fixed consideration of the $ 120.0 million upfront, the $ 15.4 million in premium related to the GSK Equity Investment and the fixed consideration related to the additional target validation research funding.

The Company allocated the estimated variable consideration relating to the target validation research to the Discovery Research Collaboration programs and the variable consideration relating to the developmental milestone to the AATD Collaboration and then allocated the fixed consideration to the performance obligations on a relative standalone selling price basis.

The Company allocated the estimated variable consideration relating to the target validation research to the Discovery Research Collaboration and the variable consideration relating to the development milestone to the AATD Collaboration and then allocated the fixed consideration to the performance obligations on a relative standalone selling price basis.

The Company determined that the GSK Collaboration Agreement did not contain a significant financing component.

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The Company will reevaluate the transaction price at the end of each reporting period, and as uncertain events are resolved or other changes in circumstances occur, the Company will adjust its estimate of the transaction price.

Under the GSK Collaboration Agreement, GSK can advance up to eight programs leveraging the Company's PRISM platform and multiple RNA-targeting modalities (RNA editing, splicing, siRNA, and antisense) with target validation work ongoing across multiple therapy areas.

GSK selected its first two programs to advance to development candidates following achievement of target validation in the three months ended June 30, 2024.

These programs utilize the Company's next generation GalNAc-siRNA format and are in hepatology.

Under the GSK Collaboration Agreement, GSK was required to provide an aggregate initiation payment of $ 12.0 million to the Company for these two oligonucleotide programs, which was received during the three months ended June 30, 2024.

The following table summarizes the allocation of the total transaction price to the identified performance obligation under the GSK Collaboration Agreement, and the amount of the transaction price unsatisfied as of December 31, 2024 (in thousands):

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Discovery Research Collaboration

(1) The Unsatisfied transaction price will be recognized over the remaining research term.

GSK Collaboration Program

(1) The Unsatisfied transaction price will be recognized over the remaining applicable research or program term.

The Company developed the estimated standalone selling price for the global license for WVE-006, under the AATD Collaboration, using a discounted cash flow model.

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The amounts received that have not yet been recognized as revenue are recorded in deferred revenue on the Company’s consolidated balance sheet.

Additional funding related to the Company’s research activities under the Discovery Research Collaboration will be recorded as accounts receivable when contractually enforceable and recorded as deferred revenue, or as revenue as the services are provided.

Additional funding related to the Company’s research activities related to Discovery Research Collaboration will be recorded as accounts receivable when contractually enforceable and recorded as deferred revenue, or as revenue as the services are provided.

During the year ended December 31, 2023 , the Company achieved a developmental milestone which pertained to the initiation of dosing in healthy volunteers in the RestorAATion clinical trial program, triggering a $ 20.0 million milestone payment to the Company from GSK.

As of December 31, 2023 , the $ 20.0 million related to the achievement of the milestone is included in the current portion of accounts receivable and payment was received from GSK in the first quarter of 2024.

During the year ended December 31, 2023, the Company recognized revenue of $ 66.3 million under the GSK Collaboration Agreement using the input method described above, which includes the $ 8.3 million of unconstrained revenue recognized for achievement of the developmental milestone mentioned above.

The aggregate amount of the transaction price allocated to the Company’s unsatisfied and partially unsatisfied performance obligations and recorded in deferred revenue on December 31, 2023 is approximately $ 94.3 million, of which approximately $ 78.7 million is included in current liabilities and $ 15.6 million is included in long-term liabilities.

As of December 31, 2023 , the $ 20.0 million related to the achievement of the milestone was included in the current portion of accounts receivable and payment was received from GSK in the first quarter of 2024.

Under the GSK Collaboration Agreement, during the years ended December 31, 2024 and December 31, 2023, the Company recognized revenue of $ 37.0 million and $ 66.3 million, respectively, using the input method described above.

The aggregate amount of the transaction price allocated to the Company’s unsatisfied and partially unsatisfied performance obligations which are recorded in deferred revenue as of December 31, 2024 is approximately $ 72.1 million, of which approximately $ 66.0 million is included in current liabilities and $ 6.1 million is included in long-term liabilities.

The aggregate amount of the transaction price allocated to the Company’s unsatisfied and partially unsatisfied performance obligations which were recorded in deferred revenue as of December 31, 2023 was approximately $ 94.3 million, of which approximately $ 78.7 million was included in current liabilities and $ 15.6 million was included in long-term liabilities.

Takeda Collaboration and Equity Agreements

In February 2018 , Wave USA and Wave UK entered into a global strategic collaboration (the “Takeda Collaboration”) with Takeda Pharmaceutical Company Limited (“Takeda”), pursuant to which Wave USA, Wave UK and Takeda agreed to collaborate on the research, development and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System (“CNS”).

The Takeda Collaboration provides the Company with at least $ 230.0 million in committed cash and Takeda with the option to co-develop and co-commercialize the Company’s CNS development programs in (1) Huntington’s disease (“HD”);

The Takeda Collaboration provided the Company with at least $ 230.0 million in committed cash and Takeda with the option to co-develop and co-commercialize the Company’s CNS development programs in (1) Huntington’s disease (“HD”);

(2) amyotrophic lateral sclerosis (“ALS”) and frontotemporal dementia (“FTD”);

and (3) the Company’s discovery-stage program targeting ATXN3 for the treatment of spinocerebellar ataxia 3 (“SCA3”) (collectively, “Category 1 Programs”).

In addition, the Takeda Collaboration

provided Takeda the right to exclusively license multiple preclinical programs for CNS disorders, including Alzheimer’s disease and Parkinson’s disease (collectively, “Category 2 Programs”).

In addition, the Takeda Collaboration provided Takeda the right to exclusively license multiple preclinical programs for CNS disorders, including Alzheimer’s disease and Parkinson’s disease (collectively, “Category 2 Programs”).

In April 2018, the Takeda Collaboration became effective and Takeda paid the Company $ 110.0 million as an upfront payment.

Takeda also agreed to fund the Company’s research and preclinical activities in the amount of $ 60.0 million during the four-year research term and to reimburse the Company for any collaboration-budgeted research and preclinical expenses incurred by Wave that exceed that amount.

Simultaneously with Wave USA and Wave UK’s entry into the collaboration and license agreement with Takeda (the “Takeda Collaboration Agreement”), the Company entered into a share purchase agreement with Takeda (the “Takeda Equity Agreement,” and together with the Takeda Collaboration Agreement, the “Takeda Agreements”) pursuant to which it agreed to sell to Takeda 1,096,892 of its ordinary shares at a purchase price of $ 54.70 per share.

Simultaneously with Wave USA and Wave UK’s entry into the collaboration and license agreement with Takeda dated February 19, 2018, as amended (the “Takeda Collaboration Agreement”), the Company entered into a share purchase agreement with Takeda (the “Takeda Equity Agreement,” and together with the Takeda Collaboration Agreement, the “Takeda Agreements”) pursuant to which it agreed to sell to Takeda 1,096,892 of its ordinary shares at a purchase price of $ 54.70 per share.

In April 2018 , the Company closed the Takeda Equity Agreement and received aggregate cash proceeds of $ 60.0 million.

The Company did not incur any material costs in connection with the issuance of the shares.

With respect to Category 1 Programs, the Company will be responsible for researching and developing products and companion diagnostics for Category 1 Programs through completion of the first proof of mechanism study for such products.

Takeda will have an exclusive option for each target and all associated products and companion diagnostics for such target, which it may exercise at any time through completion of the proof of mechanism study.

If Takeda exercises this option, the Company will receive an opt-in payment and will lead manufacturing and joint clinical co-development activities and Takeda will lead joint co-commercial activities in the United States and all commercial activities outside of the United States.

Global costs and potential profits will be shared 50 :

50 and the Company will be eligible to receive development and commercial milestone payments.

In addition to its 50% profit share, the Company is eligible to receive option exercise fees and development and commercial milestone payments for each of the Category 1 Programs.

With respect to Category 1 Programs, the Company was responsible for researching and developing products and companion diagnostics for Category 1 Programs through completion of the first proof of mechanism study for such products.

Takeda had an exclusive option for each target and all associated products and companion diagnostics for such target, which it could exercise at any time through completion of the proof of mechanism study.

If Takeda had exercised this option, the Company would have received an opt-in payment and would have led manufacturing and joint clinical co-development activities and Takeda would have led joint co-commercial activities in the United States and all commercial activities outside of the United States.

Global costs and potential profits would have been shared 50 :

50 and the Company would have been eligible to receive development and commercial milestone payments.

In addition to its 50% profit share, the Company was eligible to receive option exercise fees and development and commercial milestone payments for each of the Category 1 Programs.

With respect to Category 2 Programs, the Company granted Takeda the right to exclusively license multiple preclinical programs during a four-year research term (subject to limited extension for programs that were initiated prior to the expiration of the research term, in accordance with the Takeda Collaboration Agreement) (“Category 2 Research Term”).

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Under the Takeda Collaboration Agreement, each party granted to the other party specific intellectual property licenses to enable the other party to perform its obligations and exercise its rights under the Takeda Collaboration Agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the Takeda Collaboration Agreement.

The term of the Takeda Collaboration Agreement commenced on April 2, 2018 and, unless terminated earlier, will continue until the date on which:

The term of the Takeda Collaboration Agreement commenced on April 2, 2018 and, unless terminated earlier, would have continued until the date on which:

(i) with respect to each Category 1 Program target for which Takeda does not exercise its option, the expiration or termination of the development program with respect to such target;

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or (iii) with respect to each Category 2 Program target, the date on which royalties are no longer payable with respect to products directed to such target.

Takeda may terminate the Takeda Collaboration Agreement for convenience on 180 days ’ notice, in its entirety or on a target-by-target basis.

Subject to certain exceptions, each party has the right to terminate the Takeda Collaboration Agreement on a target-by-target basis if the other party, or a third party related to such party, challenges the patentability, enforceability or validity of any patents within the licensed technology that cover any product or companion diagnostic that is subject to the Takeda Collaboration Agreement.

In the event of any material breach of the Takeda Collaboration Agreement by a party, subject to cure rights, the other party may terminate the Takeda Collaboration Agreement in its entirety if the breach relates to all targets or on a target-by-target basis if the breach relates to a specific target.

In the event that Takeda and its affiliates cease development, manufacturing and commercialization activities with respect to compounds or products subject to the Takeda Collaboration Agreement and directed to a particular target, the Company may terminate the Takeda Collaboration Agreement with respect to such target.

Either party may terminate the Takeda Collaboration Agreement for the other party’s insolvency.

In certain termination circumstances, the Company would receive a license from Takeda to continue researching, developing and manufacturing certain products, and companion diagnostics.

The Takeda Collaboration is managed by a joint steering committee in which both parties are represented equally.

The joint steering committee is tasked with overseeing the scientific progression of each Category 1 Program and, prior to the Amendment (discussed below), the Category 2 Programs.

Takeda had the right to terminate the Takeda Collaboration Agreement for convenience on 180 days ’ notice, in its entirety or on a target-by-target basis.

Subject to certain exceptions, each party had the right to terminate the Takeda Collaboration Agreement on a target-by-target basis if the other party, or a third party related to such party, challenges the patentability, enforceability or validity of any patents within the licensed technology that cover any product or companion diagnostic that was subject to the Takeda Collaboration Agreement.

In the event of any material breach of the Takeda Collaboration Agreement by a party, subject to cure rights, the other party had the right to terminate the Takeda Collaboration Agreement in its entirety if the breach related to all targets or on a target-by-target basis if the breach related to a specific target.

In the event that Takeda and its affiliates ceased development, manufacturing and commercialization activities with respect to compounds or products subject to the Takeda Collaboration Agreement and directed to a particular target, the Company had the right to terminate the Takeda Collaboration Agreement with respect to such target.

Either party had the right to terminate the Takeda Collaboration Agreement for the other party’s insolvency.

In certain termination circumstances, the Company would have received a license from Takeda to continue researching, developing and manufacturing certain products, and companion diagnostics.

The Takeda Collaboration was managed by a joint steering committee in which both parties were represented equally.

The joint steering committee was tasked with overseeing the scientific progression of each Category 1 Program and, prior to the Amendment (discussed below), the Category 2 Programs.

The Company assessed this arrangement in accordance with ASC 606 and concluded that the contract counterparty, Takeda, is a customer for Category 1 Programs prior to Takeda exercising its option, and for Category 2 Programs during the Category 2 Research Term.

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The Company determined that the Takeda Collaboration Agreement did not contain a significant financing component.

The option exercise fees to license, co-develop and co-commercialize each Category 1 Program that may be received are excluded from the transaction price until each customer option is exercised.

The option exercise fees to license, co-develop and co-commercialize each Category 1 Program that could have been received were excluded from the transaction price until each customer option was exercised.

The potential milestone payments were excluded from the transaction price, as all milestone amounts were fully constrained at the inception of the Takeda Collaboration Agreement.

The Company will reevaluate the transaction price at the end of each reporting period and, as uncertain events are resolved or other changes in circumstances occur, if necessary, will adjust its estimate of the transaction price.

The Company would have reevaluated the transaction price at the end of each reporting period and, as uncertain events were resolved or other changes in circumstances occurred, if necessary, would have adjusted its estimate of the transaction price.

The Company allocated the transaction price to the performance obligations on a relative standalone selling price basis.

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the research and development services through completion of the first proof of mechanism and non-exclusive research and development license for SCA3;

and the research and preclinical development services and right to exclusively license the Category 2 Programs, the Company determined the standalone selling price using estimates of the costs to perform the research and development services, including expected internal and external costs for services and supplies, adjusted to reflect a profit margin.

and the research and preclinical development services and right to exclusively license the Category 2 Programs, the Company determined the

standalone selling price using estimates of the costs to perform the research and development services, including expected internal and external costs for services and supplies, adjusted to reflect a profit margin.

The total estimated cost of the research and development services reflected the nature of the services to be performed and the Company’s best estimate of the length of time required to perform the services.

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and the material right provided for the exclusive option to license, co-develop and co-commercialize SCA3, the Company estimated the standalone fair value of the option to license each Category 1 Program utilizing an adjusted market assessment approach, and determined that any standalone fair value in excess of the amounts to be paid by Takeda associated with each option represented a material right.

Revenue associated with the research and development services for each Category 1 Program performance obligation is being recognized as the research and development services are provided using an input method, according to the costs incurred on each Category 1 Program and the total costs expected to be incurred to satisfy each Category 1 Program performance obligation.

Revenue associated with the research and development services for each Category 1 Program performance obligation were recognized as the research and development services were provided using an input method, according to the costs incurred on each Category 1 Program and the total costs expected to be incurred to satisfy each Category 1 Program performance obligation.

Prior to the Amendment described below, revenue associated with the research and preclinical development services for the Category 2 Programs performance obligation was recognized as the research and preclinical development services that were provided using an input method, according to the costs incurred on Category 2 Programs and the total costs expected to be incurred to satisfy the performance obligation.

The amount allocated to the material right for each Category 1 Program option will be recognized on the date

that Takeda exercises each respective option, or immediately as each option expires unexercised.

The amounts received that have not yet been recognized as revenue are recorded in deferred revenue on the Company’s consolidated balance sheet.

The amount allocated to the material right for each Category 1 Program option would have been recognized on the date that Takeda exercised each respective option, or immediately as each option expired unexercised.

The amounts received that were not yet recognized as revenue were recorded in deferred revenue on the Company’s consolidated balance sheet.

On October 15, 2021 , Wave USA, Wave UK and Takeda entered into the Second Amendment to the Takeda Collaboration Agreement (the “Amendment”), which discontinued the Category 2 component of the Takeda Collaboration.

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As a result of the termination of the C9 for ALS/FTD Category 1 Program, the Company recognized $ 28.0 million in revenue during the three months ended September 30, 2023, which represented the remainder of the deferred revenue for the C9 for ALS/FTD Category 1 Program as of June 30, 2023.

In December 2023, the joint steering committee that manages the Takeda Collaboration, terminated SCA3 Program, another one of the Category 1 Programs, as a target under the collaboration and consequently Takeda and the Company’s rights and obligations under the Takeda Collaboration were terminated with respect to the SCA3 Category 1 Program.

As a result of the termination of the SCA3 Category 1 Program, the Company recognized $ 9.9 million in revenue during the three months ended December 31, 2023, which represented the remainder of the deferred revenue for the SCA3 Category 1 Program as of September 30, 2023.

In the third quarter of 2023, the Company achieved a developmental milestone related to the HD Category 1 Program, which pertained to the positive results from a non-clinical study of WVE-003 in non-human primates (“NHPs”).

As a result of achieving the milestone, the Company recognized $ 7.0 million in revenue, which was not previously recorded in deferred revenue, as it was fully constrained at the inception of the Takeda Collaboration.

During the years ended December 31, 2023 and 2022 , the Company recognized revenue of approximately $ 47.0 million and $ 3.3 million, respectively, under the Takeda Collaboration Agreement in the Company’s consolidated statements of operations and comprehensive loss.

Through December 31, 2023 , the Company had recognized revenue of $ 128.2 million under the Takeda Collaboration Agreement as collaboration revenue in the Company’s consolidated statements of operations and comprehensive loss.

The aggregate amount of the transaction price allocated to the Company’s unsatisfied and partially unsatisfied performance obligations and recorded in deferred revenue as of December 31, 2023 and 2022 , was $ 71.3 million and $ 111.3 million, respectively.

The deferred revenue included in current liabilities as of December 31, 2023 and 2022 , was $ 71.3 million and $ 31.6 million, respectively.

There was no deferred revenue included in long-term liabilities as of December 31, 2023, the deferred revenue included in long-term liabilities as of December 31, 2022 was $ 79.8 million.

The decrease in the long-term portion of deferred revenue as of December 31, 2023, was related to the expectation of the timing of the HD data that the Company expects to report in the second quarter of 2024, which will form the basis for decision making for the advancement of this program.

As a result, the deferred revenue related to this program was moved to short term as of December 31, 2023.

Additionally, the current portion of deferred revenue decreased from December 31, 2022 to December 31, 2023 due to revenue recognized during the year ended December 31, 2023.

The Company expects to recognize revenue for the portion of the deferred revenue that relates to the research and development services for each remaining Category 1 Program as costs are incurred, over the remaining research term.

The Company expects to recognize revenue for the portion of the deferred revenue that relates to the material right for each remaining Category 1 Program option upon Takeda’s exercise or termination of such option, or immediately as each option expires unexercised.

In December 2023, the joint steering committee that manages the Takeda Collaboration terminated the SCA3 Category 1 Program as a target under the collaboration and consequently Takeda and the Company’s rights and obligations under the Takeda Collaboration were terminated with respect to the SCA3 Category 1 Program.

In October 2024, the Company was notified by Takeda that Takeda did not intend to exercise and therefore elected to terminate its option (“Option Termination”) for the HD target under the Takeda Collaboration Agreement.

As HD was the last active collaboration target under the Takeda Collaboration Agreement, the Takeda Collaboration Agreement expired with immediate effect, and $ 70.2 million that was previously recorded as deferred revenue was recognized as revenue in the fourth quarter of 2024 related to this expiration.

During the years ended December 31, 2024, 2023, and 2022 , the Company recognized revenue of approximately $ 71.3 million, $ 47.0 million, and $ 3.3 million, respectively, under the Takeda Collaboration Agreement in the Company’s consolidated statements of operations and comprehensive loss.

Through December 31, 2024, the Company has recognized revenue of $ 199.5 million under the Takeda Collaboration Agreement as collaboration revenue in the Company’s consolidated statements of operations and comprehensive loss.

The aggregate amount of the transaction price allocated to the Company’s unsatisfied and partially unsatisfied performance obligations which are recorded in deferred revenue as of December 31, 2024 and 2023 , is $ 0.0 million and $ 71.3 million, respectively.

SHARE CAPITAL

The following represents the Company’s financing transactions during the years ended December 31, 2024, 2023, and 2022:

• The Company entered into an open market sales agreement with Jefferies LLC in May 2019, as amended in March 2020 and March 2022, for its at-the-market equity program.

During the year ended December 31, 2022 , the Company sold 458,092 ordinary shares under its at-the-market equity program for aggregate net proceeds of $ 1.2 million after deducting commissions and offering expenses.

During the year ended December 31, 2023 , the Company sold 751,688 ordinary shares under its at-the-market equity program for aggregate net proceeds of $ 3.1 million after deducting commissions and offering expenses.

• The Company entered into the Sales Agreement (as defined below) with Jefferies LLC ("Jefferies".

During the years ended December 31, 2024, 2023, and 2022 , the Company sold 2,952,591 , 751,688 , and 458,092 ordinary shares, respectively, under its "at-the-market" equity program for aggregate net proceeds of $ 20.4 million, $ 3.1 million, and $ 1.2 million, respectively, after deducting commissions and offering expenses.

• On June 16, 2022, the Company closed an underwritten offering (the “June 2022 Offering”) in which the Company issued and sold 25,464,483 of the Company’s ordinary shares at a price of $ 2.15 per share and pre-funded warrants (the “2022 Pre-Funded Warrants”) to purchase up to 7,093,656 of the Company’s ordinary shares at an offering price of $ 2.1499 per 2022 Pre-Funded Warrant, which represents the per share offering price for the ordinary shares less the $ 0.0001 per share exercise price for each 2022 Pre-Funded Warrant.

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A holder of 2022 Pre-Funded Warrants may not exercise the warrant if the holder, together with its affiliates, would beneficially own more than 19.99 % of the number of the Company’s ordinary shares outstanding or more than 19.99 % of the combined voting power of the Company’s securities outstanding immediately after giving effect to such exercise, unless and until shareholder approval is obtained.

• On December 7, 2023, the Company entered into an underwriting agreement relating to the issuance and sale in an underwritten offering of the Company’s ordinary shares.

• On December 11, 2023, the Company closed an underwritten public offering (the “December 2023 Offering” ) in which the Company issued and sold 20,000,000 of the Company's ordinary shares at a price of $ 5.00 per share.

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Subsequent to December 31, 2023, the Company received $ 14.0 million in net proceeds after deducting the underwriting discounts and commissions and offering expenses related to the December 2023 Offering.

• On September 27, 2024, the Company closed an underwritten public offering (the "September 2024 Offering") in which the Company issued and sold 23,125,001 of the Company’s ordinary shares at a price of $ 8.00 per share and pre-funded warrants (the “2024 Pre-Funded Warrants”) to purchase up to 1,875,023 of the Company’s ordinary shares at an offering price of $ 7.9999 per 2024 Pre-Funded Warrant, which represents the per share offering price for the ordinary shares less the $ 0.0001 per share exercise price for each 2024 Pre-Funded Warrant.

These 2024 Pre-Funded Warrants were recorded as a component of shareholders’ equity within additional paid-in capital.

The gross proceeds to the Company from the September 2024 Offering were $ 200.0 million before deducting underwriting discounts and commissions and other offering expenses.

The net proceeds to the Company from the September 2024 Offering were approximately $ 187.5 million, after deducting underwriting commissions and offering expenses.

The 2024 Pre-Funded Warrants are exercisable at any time after their original issuance and on or prior to the five-year anniversary of the original issuance date.

A holder of the 2024 Pre-Funded Warrants may not exercise the warrant if the holder, together with its affiliates, would beneficially own more than 4.99 % (or at the election of such holder, 9.99 % or 19.99 %) of the number of the Company’s ordinary shares outstanding or more than 4.99 % (or at the election of such holder, 9.99 % or 19.99 %) of the combined voting power of the Company’s securities outstanding immediately after giving effect to such exercise, unless and until shareholder approval is obtained.

• On October 1, 2024, the representatives of the underwriters in connection with the September 2024 Offering exercised their option in full to purchase an additional 3,750,000 ordinary shares, which increased the aggregate number of ordinary shares sold in the September 2024 Offering to 26,875,001 .

The Company’s aggregate gross proceeds from the September 2024 Offering were $ 230.0 million, before deducting underwriting discounts and commissions and offering expenses;

$ 30.0 million of which relates to the exercise of the underwriters’ option in October 2024.

• On November 12, 2024, the Company filed an automatic shelf registration statement on Form S-3ASR with the SEC for which the Company registered for sale an indeterminate amount of any combination of its ordinary shares, debt securities, warrants, rights and/or units from time to time and at prices and on terms that the Company may determine, which is referred to as the “2024 WKSI Shelf”.

The 2024 WKSI Shelf includes a prospectus covering up to an aggregate of $ 250.0 million in ordinary shares that the Company is able to issue and sell from time to time, through Jefferies acting as its sales agent, pursuant to the Open Market Sale Agreement, dated May 10, 2019, as amended by Amendment No.

1, dated as of March 2, 2020, Amendment No.

2, dated as of March 3, 2022, and Amendment No.

3, dated as of November 12, 2024, (as amended, the “Sales Agreement”), for its “at-the-market” equity program.

Features of the Series A Preferred Shares and Ordinary Shares

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The Wave Life Sciences Ltd.

2021 Equity Incentive Plan was approved by the Company’s shareholders and went into effect on August 10, 2021 and was amended effective as of August 9, 2022 and August 1, 2023 (as amended, the “2021 Plan”).

2021 Equity Incentive Plan was approved by the Company’s shareholders and went into effect on August 10, 2021 and was amended effective as of August 9, 2022, August 1, 2023, and August 6, 2024 (as amended, the “2021 Plan”).

The 2021 Plan serves as the successor to the Wave Life Sciences Ltd.

2014 Equity Incentive Plan, as amended (the “2014 Plan”), such that outstanding awards granted under the 2014 Plan continue to be governed by the terms of the 2014 Plan, but no awards may be made

under the 2014 Plan after August 10, 2021.

The aggregate number of ordinary shares authorized for issuance of awards under the 2021 Plan was originally 5,450,000 ordinary shares, and was subsequently increased to 11,450,000 and 17,950,000 in August 2022 and August 2023, respectively, plus the number of ordinary shares underlying any awards under the 2014 Plan that are forfeited, cancelled or otherwise terminated (other than by exercise or withheld by the Company to satisfy any tax withholding obligation) on or after August 10, 2021.

2014 Equity Incentive Plan, as amended (the “2014 Plan”), such that outstanding awards granted under the 2014 Plan continue to be governed by the terms of the 2014 Plan, but no awards may be made under the 2014 Plan after August 10, 2021.

The aggregate number of ordinary shares authorized for issuance of awards under the 2021 Plan was originally 5,450,000 ordinary shares, and was subsequently increased to 11,450,000, 17,950,000 , and 22,950,000 in August 2022, August 2023, and August 2024, respectively, plus the number of ordinary shares underlying any awards under the 2014 Plan that are forfeited, cancelled or otherwise terminated (other than by exercise or withheld by the Company to satisfy any tax withholding obligation) on or after August 10, 2021.

The 2021 Plan authorizes (and the 2014 Plan previously authorized) the board of directors or a committee of the board of directors to, among other things, grant non-qualified share options, restricted awards, which include restricted shares and restricted share units (“RSUs”), and performance awards to eligible employees and directors of the Company.

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3.46 % – 4.71 %

1.35 % – 4.23 %

Expected term (in years)

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Expected dividend yield

In October 2022, the compensation committee of the Company’s board of directors granted Dr.

In October 2022, the compensation committee of the Company’s board of directors (the "Compensation Committee") granted Dr.

Verdine, one of the Company’s founders and a member of the Company’s board of directors, a non-qualified share option for 163,467 ordinary shares ( “Verdine Scientific Advisory Grant”) as form of payment under Dr.

Verdine’s consulting agreement for scientific advisory services (as

described in Note 13) for the service period of October 1, 2022 through December 31, 2024, the vesting of which is subject to Dr.

Verdine’s consulting agreement for scientific advisory services (as described in Note 13) for the service period of October 1, 2022 through December 31, 2024, the vesting of which is subject to Dr.

Verdine’s continued service under the consulting agreement.

The Verdine Scientific Advisory Grant was the only non-employee grant made during the year ended December 31, 2022, and t here were no equity grants made to non-employees during the year ended December 31, 2023.

The Verdine Scientific Advisory Grant was granted as a non-employee grant during the year ended December 31, 2022, and there were no equity grants made to non-employees during the years ended December 31, 2023 and 2024.

The assumptions used in the Black-Scholes option pricing model to determine the fair value of the Verdine Scientific Advisory Grant were as follows:

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During the year ended December 31, 2022, the Company recorded share-based compensation expense of approximately $ 3.8 million related to the performance-based RSUs, which represents all of the expense related to the achievement of this performance-based RSU milestone.

During the years ended December 31, 2023 and 2022, the Company recognized share-based compensation expense of $ 1.0 million and $ 10.3 million, respectively, related to RSUs.

During the years ended December 31, 2024, 2023, and 2022 , the Company recognized share-based compensation expense of $ 0.8 million, $ 1.0 million, and $ 10.3 million, respectively, related to RSUs.

RSUs that are forfeited are available to be granted again.

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Time-based RSUs generally vest over periods of one to four years .

During the years ended December 31, 2023 and 2022, the Company recognized share-based compensation expense related to options of $ 8.5 million and $ 6.7 million, respectively.

The total intrinsic value of options exercised was $ 0.3 million and $ 0.4 million for the years ended December 31, 2023 and 2022, respectively.

During the years ended December 31, 2024, 2023, and 2022 , the Company recognized share-based compensation expense related to options of $ 11.9 million, $ 8.5 million, and $ 6.7 million, respectively.

The total intrinsic value of options exercised was $ 4.4 million, $ 0.3 million, and $ 0.4 million for the years ended December 31, 2024, 2023, and 2022, respectively.

As of December 31, 2024, the unrecognized compensation cost related to outstanding options was $ 28.9 million .

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Shares are purchased at a price equal to 85 % of the lower of the fair market value of the Company’s ordinary shares on the first business day or the last business day of an offering period.

During the years ended

December 31, 2023 and 2022 , 225,913 and 166,061 ordinary shares were issued under the ESPP, respectively.

During the years ended December 31, 2024, and 2023 , 176,498 and 225,913 ordinary shares were issued under the ESPP, respectively.

The aggregate number of ordinary shares authorized for issuance under the ESPP was originally 1,000,000 and was subsequently increased to 3,000,000 in August 2023.

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Total share-based compensation expense

Of the total share-based compensation expense recorded for the years ended December 31, 2023 and 2022 , $ 0.2 million and less than $ 0.1 million, respectively, were related to non-employee option grants, specifically the Verdine Scientific Advisory Grant, and all of the related expense is included in research and development expenses on the consolidated statements of operations and comprehensive loss.

Of the total share-based compensation expense recorded for the years ended December 31, 2024, 2023, and 2022 , $ 0.3 million, $ 0.2 million, and less than $ 0.1 million, respectively, were related to non-employee option grants, specifically the Verdine Scientific Advisory Grant, and all of the related expense is included in research and development expenses on the consolidated statements of operations and comprehensive loss.

Lease Arrangements

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Throughout the term of the lease, the Company is responsible for paying certain costs and expenses, in addition to the rent, as specified in the lease, including a proportionate share of applicable taxes, operating expenses and utilities.

As required under the terms of the lease agreement, the Company has placed restricted cash of approximately $ 2.7 million in a separate bank account as of December 31, 2023 and 2022.

As required under the terms of the lease agreement, the Company has placed restricted cash of approximately $ 2.8 million and $ 2.7 million in a separate bank account as of December 31, 2024 and 2023, respectively.

In April 2015, the Company entered into a lease agreement for an office and laboratory facility in Cambridge, Massachusetts (the “Cambridge Lease”), which commenced in October 2015 with a term of 7.5 years with a five-year renewal option to extend the lease .

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Therefore, as required by ASC 842, the Company calculated an incremental borrowing rate of 10.53 % and remeasured the right-of-use asset and the lease liabilities related to the Cambridge Lease Extension.

As a result, an

additional $ 12.0 million of operating right-of-use asset and corresponding operating lease liabilities were recorded relating to the Cambridge Lease Extension.

As a result, an additional $ 12.0 million of operating right-of-use asset and corresponding operating lease liabilities were recorded relating to the Cambridge Lease Extension.

The following table contains a summary of the lease costs recognized under ASC 842 and other information pertaining to the Company’s operating leases for the years ended December 31, 2024 and 2023:

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(in thousands)

2029 and thereafter

Total lease payments

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NET LOSS PER ORDINARY SHARE

In connection with the September 2024 Offering, the Company sold 1,875,023 2024 Pre-Funded Warrants, which are included in the total vested and exercisable pre-funded warrants (the 2022 Pre-Funded Warrants and the 2024 Pre-Funded Warrants are referred to together as the “Pre-Funded Warrants”).

As of December 31, 2024 and 2023 , there were 8,968,679 and 7,093,656 , respectively, vested and exercisable Pre-Funded Warrants outstanding to purchase ordinary shares for the exercise price of $ 0.0001 per share, provided that, unless and until the Company obtains shareholder approval for the issuance of the shares underlying the Pre-Funded Warrants, a holder will not be entitled to exercise any portion of any Pre-Funded Warrant, which, upon giving effect to such exercise, would cause (i) the aggregate number of our ordinary shares beneficially owned by the holder (together with its affiliates) to exceed, depending on the terms of the applicable Pre-Funded Warrants and in certain cases at the election of the holder, either 4.99 %, 9.99 % or 19.99 % of the number of our ordinary shares outstanding immediately after giving effect to the exercise, or (ii) the combined voting power of our securities beneficially owned by the holder (together with its affiliates) to exceed, depending on the terms of the applicable Pre-Funded Warrants and in certain cases at the election of the holder, either 4.99 %, 9.99 % or 19.99 % of the combined voting power of all of our securities then outstanding immediately after giving effect to the exercise, as such percentage ownership is determined in accordance with the terms of the applicable Pre-Funded Warrants.

The Pre-Funded Warrants are included in the weighted-average shares outstanding used in the calculation of basic net loss per share as the exercise price is negligible and the warrants are fully vested and exercisable.

Basic loss per share is computed by dividing net loss attributable to ordinary shareholders by the weighted-average number of ordinary shares outstanding.

The Company’s potentially dilutive shares, which include outstanding share options to purchase ordinary shares and RSUs, are considered to be ordinary share equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive.

The table below sets forth the computation of the Company’s basic and diluted net loss attributable to ordinary shareholders:

Year Ended December 31,

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Net loss per share, basic and diluted

As of December 31, 2023 and 2022 , there were 7,093,656 vested and exercisable Pre-Funded Warrants outstanding to purchase ordinary shares at an exercise price of $ 0.0001 per share.

The following potential ordinary shares, presented based on amounts outstanding at each period end, were excluded from the calculation of diluted net loss per share attributable to ordinary shareholders for the periods indicated because including them would have had an anti-dilutive effect:

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Options to purchase ordinary shares

Series A preferred shares

The components of loss before income taxes were as follows:

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Loss before income taxes

During the years ended December 31, 2023 and 2022, the Company recorded an income tax benefit of $ 0.7 million and an income tax provision of $ 0.7 million, respectively.

The income tax benefit for the year ended December 31, 2023 was due to a change in estimate in connection with recent U.S.

During the years ended December 31, 2024, 2023, and 2022 , the Company recorded no income tax benefit or provision , an income tax benefit of $ 0.7 million, and an income tax provision of $ 0.7 million, respectively.

The income tax benefit for the year ended December 31, 2023 was due to a change in estimate in connection with U.S.

tax guidance relating to the capitalization of research and development expenditures.

The income tax provision for the year ended December 31, 2022 was primarily due to the requirement under the Tax Cuts and Jobs Act of 2017 for taxpayers to capitalize and amortize research and development expenditures over five or fifteen years pursuant to Section 174 of the Internal Revenue Code of 1986, as amended (the “Code”).

The income tax provision for the year ended December 31, 2022 was primarily due to the requirement

under the Tax Cuts and Jobs Act of 2017 for taxpayers to capitalize and amortize research and development expenditures over five or fifteen years pursuant to Section 174 of the Internal Revenue Code of 1986, as amended (the “Code”).

The components of the benefit (provision) for income taxes were as follows:

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Operating lease right-of-use assets

Accumulated depreciation

Total deferred tax liabilities

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Increase in valuation allowance

Decrease in valuation allowance

Effect of foreign currency translation

Balance at end of year

As of December 31, 2023 , the Company had federal net operating loss carryforwards in the United States of $ 302.2 million, of which $ 301.4 million may be available to offset future income tax liabilities indefinitely, while $ 0.8 million of carryforwards that were in existence as of December 31, 2017 may offset future income tax liabilities up through 2037 .

As of December 31, 2023 , the Company had state net operating loss carryforwards of $ 54.7 million that will begin to expire in 2038 .

As of December 31, 2024 , the Company had federal net operating loss carryforwards in the United States of $ 313.7 million, of which $ 312.9 million may be available to offset future U.S.

federal taxable income indefinitely, while $ 0.8 million of carryforwards may offset future U.S.

federal taxable income through 2037 .

As of December 31, 2024 , the Company had U.S.

state net operating loss carryforwards of $ 64.9 million available to offset future U.S.

state taxable income that will begin to expire in 2038 .

As of December 31, 2024 and 2023 , the Company had U.S.

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federal income taxes and will begin to expire in 2042 .

As of December 31, 2023 and 2022 , the Company had state research and development tax credit carryforwards of approximately $ 2.6 million and $ 0.2 million,

respectively, available to offset future state income taxes and will begin to expire in 2037 , and no longer had state investment tax credit carryforwards.

As of December 31, 2023 and 2022 , the Company had net operating loss carryforwards in Japan of $ 1.4 million and $ 2.3 million, respectively, which may be available to offset future income tax liabilities and begin to expire in 2024 .

As of December 31, 2023 and 2022 , the Company had net operating loss carryforwards in Singapore of $ 122.0 million and $ 111.2 million, respectively, which may be available to offset future income tax liabilities and can be carried forward indefinitely.

As of December 31, 2023 and 2022 , the Company had net operating loss carryforwards in the United Kingdom of $ 335.7 million and $ 314.5 million, respectively, which may be available to offset future income tax liabilities and can be carried forward indefinitely.

As of December 31, 2024 and 2023 , the Company had U.S.

state research and development tax credit carryforwards of approximately $ 4.8 million and $ 2.6 million, respectively, available to offset future U.S.

state income taxes and will begin to expire in 2037 .

As of December 31, 2024 , the Company had a U.S.

orphan drug credit carryforward of $ 0.8 million available to offset future U.S.

federal income taxes that will begin to expire in 2042 .

As of December 31, 2024 and 2023 , the Company had net operating loss carryforwards in Japan of $ 0.7 million and $ 1.4 million, respectively, which may be available to offset future Japan taxable income and begin to expire in 2025 .

As of December 31, 2024 and 2023 , the Company had net operating loss carryforwards in Singapore of $ 132.7 million and $ 122.0 million, respectively, which may be available to offset future Singapore taxable income and can be carried forward indefinitely.

As of December 31, 2024 and 2023, the Company had net operating loss carryforwards in the United Kingdom (“UK”) of $ 339.5 million and $ 335.7 million, respectively, which may be available to offset future UK taxable income and can be carried forward indefinitely.

The Company has evaluated the positive and negative evidence bearing upon its ability to realize its deferred tax assets.

As of December 31, 2024, management has considered the Company’s history of cumulative net losses incurred since inception and its lack of commercialization of any products or generation of any revenue from product sales since inception and has concluded that it is more likely than not that the Company will not realize the benefits of the deferred tax assets in all jurisdictions.

Accordingly, a full valuation allowance has been established against those deferred tax assets as of December 31, 2023.

Accordingly, a full valuation allowance has been established against the Company's deferred tax assets as of December 31, 2024.

The valuation allowance increased by $ 25.1 million in 2024.

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Any release of valuation allowance will be recorded as a tax benefit either increasing net income or decreasing net loss.

The Company’s reserves related to taxes and its accounting for uncertain tax positions are based on a determination of whether and how much of a tax benefit taken by the Company in its tax filings or positions is more-likely-than-not to be realized following resolution of any potential contingencies present related to the tax benefit.

A summary of activity in the Company’s unrecognized tax benefits is as follows:

The Company’s reserves related to income taxes and its accounting for uncertain tax positions are based on a determination of whether and how much of a tax benefit taken by the Company in its tax filings or positions is more-likely-than-not to be realized following resolution of any potential contingencies present related to the tax benefit.

A summary of activity in the Company’s gross unrecognized tax benefits, excluding interest and penalties, is as follows:

(in thousands)

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As of December 31, 2024 and 2023 , the total amount of gross unrecognized tax benefits, which excludes interest and penalties, was $ 20.8 million and $ 15.8 million, respectively.

At December 31, 2023 , no ne of the net unrecognized tax benefits would affect the Company’s annual effective tax rate if recognized.

At December 31, 2024 , no ne of the net unrecognized tax benefits would affect the Company’s effective tax rate due to the Company's full valuation allowance.

The Company anticipates that $ 2.6 million of the total unrecognized tax benefits at December 31, 2024 will decrease within the next twelve months due to certain tax return filings.

The Company files income tax returns as prescribed by the tax laws of the jurisdictions in which it operates.

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To the extent that the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the tax authorities to the extent utilized in a future period.

As of December 31, 2023 and 2022 , $ 23.9 million and $ 44.1 million, respectively, of cash and cash equivalents were held by the subsidiaries outside of Singapore.

The Company does not provide for Singapore income tax or foreign withholding taxes on foreign unrepatriated earnings, as the Company intends to permanently reinvest undistributed earnings in its foreign subsidiaries.

If the Company decides to change this assertion in the future to repatriate any additional foreign earnings, the Company may be required to accrue and pay taxes.

Because of the complexity of Singapore and foreign tax rules applicable to the distribution of earnings from foreign subsidiaries to Singapore, the determination of the unrecognized deferred tax liability on these earnings is not practicable.

As of December 31, 2024 and 2023 , $ 37.8 million and $ 23.9 million, respectively, of cash and cash equivalents were held by the Company’s subsidiaries outside of Singapore.

The Company does not provide for Singapore income tax or withholding taxes on the outside basis differences, including foreign unremitted earnings of its subsidiaries as they are permanently reinvested.

If the Company decides to change its indefinite reversal assertion in the future, the Company may be required to record deferred taxes.

Because of the complexity of Singapore and the rest-of-the-world tax rules applicable to the method of recovery of the investment in its subsidiaries, including distribution of earnings from its subsidiaries to Singapore, the determination of the unrecognized deferred tax liability is not practicable.

Utilization of the net operating loss carryforwards and research and development tax credit carryforwards in the United States may be subject to a substantial annual limitation under Section 382 of the Code, due to ownership changes that have occurred previously or that could occur in the future.

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In general, an ownership change, as defined by Section 382, results from transactions increasing the ownership of certain shareholders or public groups in the shares of a corporation by more than 50% over a three-year period.

In 2018, 2020 and 2022, the Company completed studies to assess whether an ownership change had occurred or whether there have been multiple ownership changes since its formation.

The results of the 2018 and 2020 studies indicated that the Company experienced ownership

changes as defined by Section 382 of the Code.

Based on the results of the 2018 and 2020 studies, management had determined that the limitations will not have a material impact on the Company’s ability to utilize its net operating losses and research and development credit carryforwards to offset future tax liabilities.

The results of the 2022 study indicated that the Company experienced an ownership change and will be limited in its ability to utilize net operating losses and its research and development credit carryforwards in the 2022 tax year and going forward.

The Company has completed Section 382 studies to assess whether there have been ownership changes since its formation through 2022.

The results of the studies indicated that the Company experienced ownership changes as defined by Section 382 of the Code, and, as such, the Company has adjusted its net operating losses and research and development credit carryforwards to reflect the limitations as a result of such ownership changes.

Should one or more ownership changes occur in the future, the Company’s ability to utilize its net operating losses and research and development credit carryforwards may be further limited.

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Verdine approximately $ 13 thousand per month, plus reimbursement for certain expenses.

In October 2022, the compensation committee of the Company’s board of directors granted Dr.

In October 2022, the Compensation Committee granted Dr.

Verdine a non-qualified share option for 163,467 ordinary shares in lieu of cash as payment under this consulting agreement for the service period of October 1, 2022 through December 31, 2024, the monthly vesting of which is subject to Dr.

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(“SNBL”), one of the Company’s shareholders, to provide approximately $ 2.8 million in certain NHPs contract research services to the Company.

During the year ended December 31, 2023 , the Company paid SNBL $ 1.4 million for the aforementioned contract research services.

During the years ended December 31, 2024 and 2023 , the Company made payments of $ 0.9 million and $ 1.4 million, respectively, to SNBL.

Through December 31, 2024 , the Company has paid $ 2.3 million to SNBL for the aforementioned NHP contract research services.

SEGMENT INFORMATION

Operating segments are defined as components of an entity for which separate financial information is available and that is regularly reviewed by the CODM in deciding how to allocate resources to an individual segment and in assessing performance.

The Company operates as a single reporting segment, focused on developing its proprietary RNA medicines platform, PRISM, to develop and commercialize a broad pipeline of RNA medicines in a variety of therapeutic areas.

Consistent with our operational structure, our CEO, as the CODM , manages and allocates resources on a consolidated basis at the global corporate level.

The results of our operations are reported on a consolidated basis for purposes of segment reporting.

The CEO uses consolidated net loss that is reported on the consolidated statements of operations and comprehensive loss for the purposes of assessing performance, allocating resources and planning, monitoring budget versus actual results, and forecasting future periods.

The following table is representative of the significant expense categories regularly provided to the CODM when managing the Company's single reporting segment.

A reconciliation to consolidated operating expenses as our single segment operating loss for the years ended December 31, 2024, 2023, and 2022 is included in the table below:

For the Year Ended December 31,

(in thousands)

Research and development expenses:

Other research and development expenses (1) , including INHBE, RNA editing, PRISM, others

ALS and FTD programs (discontinued)

Total research and development expenses

General and administrative expenses

Total operating expenses

Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 7. Management’s Discussion and Analysis