What changed in this filing

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Item 1. Financial Statements

2026-08-06 compared with 2026-05-07 · 166 added, 97 removed, 530 unchanged (33% of the section changed)

2 unchanged sentences

CONDENSED CONSOLIDATED BALANCE SHEETS

(in thousands, except share and per share amounts) March 31,

(in thousands, except share and per share amounts) June 30,

2026 December 31,

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775,000,000 shares authorized;

180,615,514 and 180,514,014 shares issued and outstanding as of March 31, 2026 and December 31, 2025, respectively

180,737,548 and 180,514,014 shares issued and outstanding as of June 30, 2026 and December 31, 2025, respectively

Additional paid-in capital 1,019,401 1,009,138

7 unchanged sentences

Three Months Ended

June 30, Six Months Ended

(in thousands, except share and per share amounts) 2026 2025 2026 2025

31 unchanged sentences

Stock-based compensation expense — — 4,644 — — 4,644

Change in unrealized income (loss) on investments, net of tax — — — ( 83 ) — ( 83 )

Unrealized loss on investments during the period, net of tax — — — ( 83 ) — ( 83 )

Net loss — — — — ( 49,122 ) ( 49,122 )

Balance as of March 31, 2026 180,615,514 $ 2 $ 1,014,175 $ ( 1 ) $ ( 937,476 ) $ 76,700

Issuance of common stock under the employee stock purchase plan 112,110 — 338 — — 338

Issuance of common stock upon exercise of stock options 9,924 — 23 — — 23

Stock-based compensation expense — — 4,865 — — 4,865

Unrealized loss on investments during the period, net of tax — — — ( 8 ) — ( 8 )

Net loss — — — — ( 42,109 ) ( 42,109 )

Balance as of June 30, 2026 180,737,548 $ 2 $ 1,019,401 $ ( 9 ) $ ( 979,585 ) $ 39,809

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

10 unchanged sentences

Stock-based compensation expense — — 5,681 — — 5,681

Change in unrealized income (loss) on investments, net of tax — — — ( 16 ) — ( 16 )

Unrealized loss on investments during the period, net of tax — — — ( 16 ) — ( 16 )

Net loss — — — — ( 51,890 ) ( 51,890 )

Balance as of March 31, 2025 131,160,842 $ 2 $ 846,039 $ ( 15 ) $ ( 739,102 ) $ 106,924

Exercise of pre-funded warrants 3,199,984 — — — — —

Issuance of common stock under the employee stock purchase plan 133,612 — 197 — — 197

Stock-based compensation expense — — 6,325 — — 6,325

Unrealized gain on investments during the period, net of tax — — — 5 — 5

Net loss — — — — ( 52,591 ) ( 52,591 )

Balance as of June 30, 2025 134,494,438 $ 2 $ 852,561 $ ( 10 ) $ ( 791,693 ) $ 60,860

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

1 unchanged sentence

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Three Months Ended

Six Months Ended

(in thousands)

22 unchanged sentences

Net proceeds from stock option exercises 416 —

Proceeds from ESPP offerings 338 197

Proceeds from research and development funding liability — 6,000

6 unchanged sentences

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

Three Months Ended

Six Months Ended

(in thousands)

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To date, the Company has funded its operations primarily with proceeds from sales of preferred stock, from public offerings, and private placement of its common stock, and through payments from its collaboration partners.

Since its inception, the Company has incurred substantial losses and, as of March 31, 2026, the Company had an accumulated deficit of $ 937.5 million.

Since its inception, the Company has incurred substantial losses and, as of June 30, 2026, the Company had an accumulated deficit of $ 979.6 million.

The Company expects to generate operating losses and negative operating cash flows for the foreseeable future.

As of March 31, 2026, the Company maintains cash, cash equivalents, and short-term investments of $ 135.5 million.

As of June 30, 2026, the Company maintains cash, cash equivalents, and short-term investments of $ 95.1 million.

Going Concern

In accordance with Accounting Standards Codification, or ASC, 205-40, Going Concern , or ASC 205-40, the Company evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about the Company’s ability to continue as a going concern within one year after the date on which this Quarterly Report on Form 10-Q is filed.

Based on the Company's cash, cash equivalents, and short-term investments as of March 31, 2026, the Company's current and forecasted level of operations, and its forecasted cash flows, the Company’s ability to continue as a going concern is dependent upon its ability to obtain the necessary financing to meet its obligations and repay its liabilities arising from normal business operations when they come due.

Based on the Company's cash, cash equivalents, and short-term investments as of June 30, 2026, the Company's current and forecasted level of operations, and its forecasted cash flows, the Company’s ability to continue as a going concern is dependent upon its ability to obtain the necessary financing to meet its obligations and repay its liabilities arising from normal business operations when they come due.

Management plans to provide for the Company’s capital requirements through financing or other transactions, and selling shares under the Company's “at the market offering” program.

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As a result, management has concluded that substantial doubt exists about the Company’s ability to continue as a going concern for 12 months from the date these condensed consolidated financial statements are issued.

The condensed consolidated financial statements as of March 31, 2026 have been prepared under the assumption that the Company will continue as a going concern for the next 12 months and that contemplates the realization of assets and satisfaction of liabilities and commitments in the normal course of business.

The condensed consolidated financial statements as of June 30, 2026 have been prepared under the assumption that the Company will continue as a going concern for the next 12 months and that contemplates the realization of assets and satisfaction of liabilities and commitments in the normal course of business.

The Company’s ability to continue as a going concern is dependent upon its uncertain ability to obtain additional capital, reduce expenditures and/or execute on its business plan.

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are unaudited.

The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited annual consolidated financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s financial position as of March 31, 2026, the results of its operations for the three months ended March 31, 2026 and 2025, the condensed consolidated statements of stockholders’ equity for the three months ended March 31, 2026 and 2025, and its cash flows for the three months ended March 31, 2026 and 2025.

The financial data and other information disclosed in these notes related to the three months ended March 31, 2026 and 2025 are also unaudited.

The results for the three months ended March 31, 2026 are not necessarily indicative of results to be expected for the year ending December 31, 2026, any other interim periods, or any future year or period.

The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited annual consolidated financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s financial position as of June 30, 2026, the results of its operations for the three and six months ended June 30, 2026 and 2025, the condensed consolidated statements of stockholders’ equity for the three and six months ended June 30, 2026 and 2025, and its cash flows for the six months ended June 30, 2026 and 2025.

The financial data and other information disclosed in these notes related to the three and six months ended June 30, 2026 and 2025 are also unaudited.

The results for the three and six months ended June 30, 2026 are not necessarily indicative of results to be expected for the year ending December 31, 2026, any other interim periods, or any future year or period.

The condensed consolidated balance sheet data as of December 31, 2025 was derived from our audited financial statements, but does not include all disclosures required by U.S.

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The Company’s significant accounting policies are disclosed in Note 2, Summary of significant accounting policies , in the audited consolidated financial statements for the year ended December 31, 2025, and notes thereto, included in the Company’s Annual Report on Form 10-K that was filed with the SEC on March 3, 2026.

Since the date of those financial statements, there have been no material changes to its significant accounting policies, except as noted below.

Since the date of those financial statements, there have been no material changes to its significant accounting policies.

Use of Estimates

The preparation of the Company’s condensed consolidated financial statements in conformity with U.S.

GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the condensed consolidated financial statements

and the reported amounts of revenues and expenses during the reporting periods.

Significant estimates and assumptions reflected within these condensed consolidated financial statements include, but are not limited to, the valuation of the Company’s common stock and stock-based awards.

Significant estimates and

assumptions reflected within these condensed consolidated financial statements include, but are not limited to, the valuation of the Company’s common stock and stock-based awards.

The Company bases its estimates on historical experience, known trends and other market-specific or other relevant factors that it believes to be reasonable under the circumstances.

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The CODM performs this assessment based on the Company’s consolidated net loss.

Through this analysis, the CODM assesses performance by comparing actual net loss verses the budget, and then decides how to allocate resources to invest in the Company’s research and development programs.

Through this analysis, the CODM assesses performance by comparing actual net loss against the budget, and then decides how to allocate resources to invest in the Company’s research and development programs.

The measure of segment assets is reported on the condensed consolidated balance sheets as total assets.

1 unchanged sentence

Three Months Ended

June 30, Six Months Ended

(in thousands) 2026 2025 2026 2025

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Other segment items (1)

11,345 11,081 28,744 20,966

Total operating expenses 44,439 54,492 95,949 108,338

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The amendments can be applied prospectively or modified retrospectively and are effective for annual and interim periods beginning after December 15, 2026.

The Company is currently evaluating the impact to the Company’s consolidated financial statements.

The Company does not expect this ASU to have a material impact on its consolidated financial statements.

In December 2025, the FASB issued ASU No.

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The following tables present the Company’s fair value hierarchy for its assets that are measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair value:

As of March 31, 2026:

As of June 30, 2026:

(in thousands) Level 1 Level 2 Level 3 Total

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Unrealized gains and losses of investments in debt securities consisted of the following:

As of March 31, 2026:

As of June 30, 2026:

(in thousands) Amortized Cost Unrealized Gains Unrealized Losses Fair Value

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The contractual maturities of the Company’s investments in debt securities were as follows:

(in thousands) March 31,

(in thousands) June 30,

2026 December 31,

Due within one year $ 47,620 $ 114,648

Marketable securities in unrealized loss positions consisted of the following:

As of March 31, 2026:

Investments in unrealized loss positions consisted of the following:

As of June 30, 2026:

(in thousands, except number of securities) Number of Securities Fair Value Gross Unrealized Losses

2 unchanged sentences

Corporate debt securities 13 $ 11,550 $ ( 5 )

Based on factors such as historical experience, market data, issuer-specific factors, and current economic conditions, the Company did not record an allowance for credit losses as of March 31, 2026 related to these investments.

Further, given the lack of significant change in the credit risk, the Company does not consider these investments to be impaired.

1 unchanged sentence

Property and equipment, net consisted of the following:

(in thousands) March 31,

(in thousands) June 30,

2026 December 31,

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Depreciation expense related to property and equipment is as follows:

Three Months Ended March 31,

Three Months Ended June 30, Six Months Ended June 30,

(in thousands) 2026 2025 2026 2025

2 unchanged sentences

Accrued expenses and other current liabilities consisted of the following:

(in thousands) March 31,

(in thousands) June 30,

2026 December 31,

Accrued expenses and other current liabilities

Research and clinical costs

$ 2,197 $ 1,997

Employee compensation and benefits $ 3,415 $ 7,337

License fee 3,312 1,562

Research and clinical costs

Other 916 1,704

3 unchanged sentences

Since the date of those financial statements, there have been no changes to the Company’s significant agreements.

The table below reconciles the undiscounted future annual lease payments to the total operating lease liabilities recorded in the condensed consolidated balance sheet as of March 31, 2026:

The table below reconciles the undiscounted future annual lease payments to the total operating lease liabilities recorded in the condensed consolidated balance sheet as of June 30, 2026:

(in thousands) Undiscounted

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Total operating lease liability $ 112,478

Stockholder’s Equity

Under the Company’s Third Amended and Restated of Certificate of Incorporation, as amended, the Company’s common stock had a par value of $ 0.00001 and each share of common stock entitles the holder to one vote on all matters submitted to the stockholders for a vote.

Stockholders’ Equity

Under the Company’s Third Amended and Restated Certificate of Incorporation, as amended, the Company’s common stock had a par value of $ 0.00001 and each share of common stock entitles the holder to one vote on all matters submitted to the stockholders for a vote.

The holders of common stock are entitled to receive dividends, if any, as declared by the Company’s Board of Directors, or the Board of Directors.

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The common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary.

As of March 31, 2026 and as of the date these condensed consolidated financial statements are issued, the Company has not sold any shares of common stock under the ATM Prospectus.

Stock-Based Compensation

1 unchanged sentence

The Company’s 2019 Stock Option and Grant Plan, or the 2019 Plan, provided for the Company to grant incentive stock options, non-qualified stock options, unrestricted stock awards, restricted stock awards and other stock-based awards to the officers, employees, consultants and other key persons of the Company.

In October 2022, in

connection with the closing of the Company’s initial public offering, the Board of Directors determined that no further awards would be granted under the 2019 Plan.

In October 2022, in connection with the closing of the Company’s initial public offering, the Board of Directors determined that no further awards would be granted under the 2019 Plan.

2022 Stock Option and Incentive Plan

5 unchanged sentences

On January 1, 2026, the annual increase resulted in an additional 9,025,700 shares authorized being added to the 2022 Plan.

As of March 31, 2026, the Company had 34,482,330 shares reserved under the 2022 Plan and the 2019 Plan, and 11,573,377 shares available for issuance under the 2022 Plan.

As of June 30, 2026, the Company had 34,772,406 shares reserved under the 2022 Plan and the 2019 Plan, and 10,808,408 shares available for issuance under the 2022 Plan.

2022 Employee Stock Purchase Plan

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There was no annual increase for the 2022 ESPP on January 1, 2026.

As of March 31, 2026, the Company had 1,522,025 shares available for issuance under the 2022 ESPP.

During the three months ended March 31, 2026, the Company issued no shares of the Company’s common stock under the 2022 ESPP.

As of June 30, 2026, the Company had 1,409,915 shares available for issuance under the 2022 ESPP.

During the six months ended June 30, 2026, the Company issued 112,110 shares of the Company’s common stock under the 2022 ESPP.

Stock Options

The following table summarizes the Company’s time-based stock option activity for the three months ended March 31, 2026:

The following table summarizes the Company’s time-based stock option activity for the six months ended June 30, 2026:

Number of Shares Weighted-Average

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Cancelled or forfeited ( 962,560 ) 5.86

Outstanding at March 31, 2026 20,879,389 $ 3.71

Options vested and exercisable at March 31, 2026 7,875,474 $ 4.69

Options vested and expected to vest at March 31, 2026 20,879,389 $ 3.71

As of March 31, 2026, there was $ 36.5 million of total unrecognized compensation cost related to time-based stock options, including incremental expense related to the Company’s option repricing announced in August 2025.

Outstanding at June 30, 2026 21,734,434 $ 3.60

Options vested and exercisable at June 30, 2026 9,420,268 $ 4.20

Options vested and expected to vest at June 30, 2026 21,734,434 $ 3.60

As of June 30, 2026, there was $ 33.7 million of total unrecognized compensation cost related to time-based stock options, including incremental expense related to the Company’s option repricing announced in August 2025.

The Company expects to recognize such amount over a remaining weighted-average period of 2.8 years.

Performance-Based Stock Options

The following table summarizes the Company’s performance-based stock option activity for the three months ended March 31, 2026:

The following table summarizes the Company’s performance-based stock option activity for the six months ended June 30, 2026:

Number of Shares Weighted-Average

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Outstanding at December 31, 2025 2,029,564 $ 4.10

Exercised — —

Granted 100,000 3.39

Cancelled or forfeited ( 200,000 ) 8.32

Outstanding at March 31, 2026 2,029,564 $ 4.10

Vested and exercisable at March 31, 2026 813,480 $ 5.26

As of March 31, 2026, there was $ 3.1 million of total unrecognized compensation cost related to performance-based stock options.

Outstanding at June 30, 2026 1,929,564 $ 3.63

Vested and exercisable at June 30, 2026 813,480 $ 5.26

As of June 30, 2026, there was $ 2.2 million of total unrecognized compensation cost related to performance-based stock options.

Stock-Based Compensation

1 unchanged sentence

Three Months Ended

June 30, Six Months Ended

(in thousands)

2026 2025 2026 2025

Stock-based compensation expense:

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Supplemental information related to the Research Collaboration and License Agreement, or the BMS Collaboration Agreement, with Juno Therapeutics, Inc., a wholly-owned subsidiary of the Bristol-Myers Squibb Company, or BMS, consisted of the following:

(in thousands) March 31,

(in thousands) June 30,

2026 December 31,

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Three Months Ended

June 30, Six Months Ended

(in thousands) 2026 2025 2026 2025

Revenue recognized that was included in contract liability at the beginning of the period $ 1,152 $ 1,061 $ 2,008 $ 2,502

As of March 31, 2026, the aggregate amount of the transaction price allocated to performance obligations under the BMS Collaboration Agreement that are partially unsatisfied was $ 65.1 million.

Revenue recognized from performance obligations fully or partially satisfied in previous periods $ 3 $ 5 $ 3 $ 3

As of June 30, 2026, the aggregate amount of the transaction price allocated to performance obligations under the BMS Collaboration Agreement that are partially unsatisfied was $ 64.0 million.

The Company recognizes the portion of the transaction price as the single performance obligation is satisfied, using an input method, in proportion to costs incurred to date as compared to total costs incurred and expected to be incurred in the future to satisfy the underlying obligation.

Beam Collaboration Agreement — Related Party

The Company is currently engaged in arbitration proceedings with Beam Therapeutics, Inc., or Beam, regarding the parties’ collaboration and license agreement, or the Agreement.

The Company was engaged in binding arbitration proceedings with Beam Therapeutics, Inc., or Beam, regarding the parties’ collaboration and license agreement, or the Beam Collaboration Agreement.

A dispute arose between the parties following the Company’s March 18, 2025 announcement that it is developing a Prime Editing-based treatment for alpha-1 antitrypsin deficiency, or AATD.

On April 16, 2025, Beam filed an arbitration demand with the American Arbitration Association, or AAA, alleging that the Company has breached the Agreement by developing a product for the treatment of AATD and by allegedly not complying with certain obligations to transfer technical information to Beam pursuant to the Agreement.

On April 16, 2025, Beam filed an arbitration demand with the American Arbitration Association, or AAA, alleging that the Company has breached the Beam Collaboration Agreement by developing a product for the treatment of AATD and by allegedly not complying with certain obligations to transfer technical information to Beam pursuant to the Beam Collaboration Agreement.

Beam also made related claims for trade secret misappropriation and various business torts based on similar allegations.

Beam seeks both declaratory, injunctive, and monetary relief.

On April 18, 2025, the Company filed an arbitration demand with the AAA seeking a declaration that the Company’s AATD program is within our “Field” as defined by the Agreement.

Beam sought both declaratory, injunctive, and monetary relief.

On April 18, 2025, the Company filed an arbitration demand with the AAA seeking a declaration that the Company’s AATD program is within our “Field” as defined by the Beam Collaboration Agreement.

The arbitrations were consolidated and a hearing was conducted earlier this year.

The Company is currently waiting for the arbitrators’ decision and final resolution.

If the final resolution of the matter is adverse to the Company, the arbitration panel may provide Beam with relief including, among other things, monetary damages and/or an order that the Company cease work on its AATD program and transfer such program to Beam.

On July 6, 2026, the Company received a final award, or the Final Award, from the arbitration tribunal, or the Tribunal.

In the Final Award, the Tribunal declared that PM647, the Company's development candidate for the treatment of AATD, is within the Company's “Field,” and that the Company therefore did not breach the Beam Collaboration Agreement.

Consequently, the Tribunal denied Beam's requests for damages and injunctive relief based on Beam's assertion that Prime breached the Beam Collaboration Agreement.

The Tribunal denied the remaining claims brought by Beam and the Company.

Net Loss per Share

1 unchanged sentence

Three Months Ended

June 30, Six Months Ended

(in thousands, except share and per share amounts) 2026 2025 2026 2025

3 unchanged sentences

Diluted net loss per share available to common stockholders is computed by giving effect to all potentially dilutive common stock equivalents outstanding for the period.

For purposes of this calculation, outstanding time-based stock options to purchase common stock, vested performance-based stock options to purchase common stock, and unvested performance-based restricted stock awards were considered common stock equivalents but had been excluded from the calculation of diluted net loss per share available to common stockholders as their effect was anti-dilutive.

For purposes of this calculation, outstanding time-based stock options to purchase common stock, vested performance-based stock options to purchase common stock, and

unvested performance-based restricted stock awards were considered common stock equivalents but had been excluded from the calculation of diluted net loss per share available to common stockholders as their effect was anti-dilutive.

In periods in which the Company reports a net loss available to common stockholders, diluted net loss per share available to common stockholders is the same as basic net loss per share available to common stockholders, since dilutive common shares are not assumed to have been issued if their effect is anti-dilutive.

The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:

As of March 31,

As of June 30,

Anti-dilutive common stock equivalents:

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We are advancing our in vivo programs to cure two of the largest genetic liver diseases, Wilson disease and AATD.

Both programs are currently in late stages of pre-clinical development and are on track for IND and/or Clinical Trial Application filings in the first half of 2026 for Wilson Disease and the middle of 2026 for AATD.

In June 2026, we received clearance of the CTA for PM577a, an investigational Prime Editor for Wilson disease, in New Zealand, representing the first clinical authorization for one of our in vivo Prime Editing therapies and enabling the initiation of our global Phase 1/2 clinical trial.

In July 2026, the FDA cleared our IND application for PM577a.

The IND clearance opens participation in our global Phase 1/2 clinical trial to patients in the United States, where H1069Q is the single most common pathogenic variant causing Wilson disease.

Additionally, we are on track for IND application submission and/or CTA filing in the third quarter of 2026 for PM647 in AATD.

We intend to leverage the modularity of our platform to expeditiously and efficiently develop these programs supported by our universal liver lipid nanoparticle along with potential regulatory, clinical and other synergies from our modular technology.

We also continue to advance our i n vivo Cystic Fibrosis program with support from Cystic Fibrosis Foundation and our efforts to develop Prime Edited CAR-T products for hematology, immunology, and oncology in partnership with BMS.

We also continue to advance our in vivo Cystic Fibrosis program with support from Cystic Fibrosis Foundation and our efforts to develop Prime Edited CAR-T products for hematology, immunology, and oncology in partnership with BMS.

In addition, we will continue to pursue additional business development opportunities to accelerate innovation, ensure the broadest application of Prime Editing, and further bolster our financial resources.

In August 2025, we announced additional data from the first patient dosed and initial data from the second patient dosed in our Phase 1/2 trial in CGD.

We continue to engage in regulatory dialogue with the FDA toward a potential Biologics License Application filing for PM359.

We continue to engage in regulatory dialogue with the FDA toward a Biologics License Application submission for PM359, which is planned for the first half of 2027.

In June 2026, the FDA granted RMAT designation for PM359, which provides opportunities for intensive FDA guidance and potential expedited review during the program’s development.

Components of Our Results of Operations

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Results of Operations

Comparison of the Three Months Ended March 31, 2026 and 2025

Comparison of the Three Months Ended June 30, 2026 and 2025

Operating Expenses

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Total research and development expenses $ 33,397 $ 41,375 $ (7,978)

The $6.5 million decrease in research and development expenses for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily driven by:

• $3.8 million decrease in research costs due to the deprioritization of our CGD programs as we strategically focus our internal efforts on advancing our in vivo liver franchise;

• $3.7 million decrease in personnel expenses, driven primarily by fewer R&D personnel resulting from the workforce reduction announced in May 2025.

The $8.0 million decrease in research and development expenses for the three months ended June 30, 2026 as compared to the three months ended June 30, 2025 was primarily driven by:

• $3.6 million decrease in personnel expenses, driven primarily by fewer personnel-related costs resulting from the workforce reduction announced in May 2025;

• $3.2 million decrease in facility costs primarily related to cost savings realized from bringing our vivarium in-house;

• $2.0 million decrease in research costs as we advance our in vivo liver franchise from research towards IND application submission and potential clinical trials;

• $1.1 million decrease in license and IP costs primarily due to license fees recognized in the second quarter of 2025.

These were offset by a $1.5 million increase in clinical expenses as we advance our Wilson disease and AATD programs.

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General and administrative expenses:

Professional and consultant fees $ 8,945 $ 3,272 $ 5,673

Personnel expenses $ 5,559 $ 7,082 $ (1,523)

Facility related and other 2,983 2,079 904

Professional and consultant fees 2,500 3,956 (1,456)

Total general and administrative expenses $ 11,042 $ 13,117 (2,075)

The $4.1 million increase in general and administrative expenses for the three months ended March 31, 2026 as compared to the three months ended March 31, 2025 was primarily driven by a $5.7 million increase in professional and consultant fees, a majority of which are arbitration-related legal expenses.

This was offset by a $1.6 million decrease in personnel expenses driven primarily by fewer G&A personnel resulting from the workforce reduction announced in May 2025.

The $2.1 million decrease in general and administrative expenses for the three months ended June 30, 2026 as compared to the three months ended June 30, 2025 was primarily driven by:

• $1.5 million decrease in personnel expenses, driven primarily by fewer personnel-related costs resulting from the workforce reduction announced in May 2025 and a decrease in stock compensation expense of $0.8 million;

• $1.5 million decrease in professional and consultant fees primarily related to lower corporate legal fees.

Other Income (Expense)

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Total other income, net $ 1,176 $ 786 $ 390

Comparison of the Six Months Ended June 30, 2026 and 2025

Operating Expenses

Research and Development Expenses

Six Months Ended

(in thousands) 2026 2025 Change

Research and development expenses:

Personnel expenses $ 21,853 $ 29,248 $ (7,395)

Facility related 20,843 24,734 (3,891)

Research costs 13,418 19,152 (5,734)

Clinical expenses 4,468 1,747 2,721

Professional and consultant fees 3,920 2,819 1,101

License, intellectual property fees, and other 3,000 4,237 (1,237)

Total research and development expenses $ 67,502 $ 81,937 $ (14,435)

The $14.4 million decrease in research and development expenses for the six months ended June 30, 2026 as compared to the six months ended June 30, 2025 was primarily driven by:

• $7.4 million decrease in personnel expenses, driven primarily by fewer personnel-related costs resulting from the workforce reduction announced in May 2025;

• $5.7 million decrease in research costs as we advance our in vivo liver franchise from research towards IND application submission and potential clinical trials;

• $3.9 million decrease in facility costs primarily related to cost savings realized from bringing our vivarium in-house and due to moving expensed incurred in 2025;

• $1.2 million decrease in license and IP costs primarily due to license fees recognized in the second quarter of 2025.

These were offset by:

• $2.7 million increase in clinical expenses as we advance our Wilson disease and AATD programs;

• $1.1 million increase in professional and consultant fees related to our in-house vivarium.

General and Administrative Expenses

Six Months Ended

(in thousands) 2026 2025 Change

General and administrative expenses:

Professional and consultant fees $ 11,445 $ 7,228 $ 4,217

Personnel expenses 11,091 14,238 (3,147)

Facility related and other 5,911 4,935 976

Total general and administrative expenses $ 28,447 $ 26,401 $ 2,046

The $2.0 million increase in general and administrative expenses for the six months ended June 30, 2026 as compared to the six months ended June 30, 2025 was primarily driven by a $4.2 million increase in professional and consultant fees, a majority of which are arbitration-related legal expenses.

This was offset by a $3.1 million decrease in personnel expenses driven primarily by fewer personnel-related costs resulting from the workforce reduction announced in May 2025 and a decrease in stock compensation expense of $1.4 million.

Other Income (Expense)

Six Months Ended

(in thousands) 2026 2025 Change

Other income:

Interest income $ 1,809 $ 1,925 $ (116)

Accretion (amortization) of investments 793 869 (76)

Change in fair value of short-term investment — related party — (1,561) 1,561

The change in fair value of related party short-term investment for the three months ended March 31, 2025 was the result of Beam’s stock price movement.

Other income, net 106 55 51

Total other income, net $ 2,708 $ 1,288 $ 1,420

Change in Fair Value of Short-Term Investment — Related Party

The change in fair value of related party short-term investment for the six months ended June 30, 2025 was the result of Beam’s stock price movement.

Liquidity and Capital Resources

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To date, we have funded our operations primarily with proceeds from sales of preferred stock and from our public offerings and through payments from our collaboration partners.

As of March 31, 2026, we had cash, cash equivalents, and investments of $135.5 million, excluding our restricted cash, or $149.2 million, including restricted cash.

As of June 30, 2026, we had cash, cash equivalents, and investments of $95.1 million, excluding our restricted cash, or $108.8 million, including restricted cash.

In March 2026, we converted our automatic shelf registration statement on Form S-3ASR (File No.

333-291348), originally filed with the SEC on November 7, 2025, to a non-automatic shelf registration statement on Form S-3, or the Registration Statement, by post-effective amendments, for the issuance and sale of up to $500.0 million of our common stock, preferred stock, debt securities, warrants and/or units or any combination thereof.

common stock, preferred stock, debt securities, warrants and/or units or any combination thereof.

The Registration Statement was declared effective by the SEC on March 4, 2026.

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Our common stock will be sold at prevailing market prices at the time of the sale, and as a result, prices may vary.

As of March 31, 2026, we have not sold any shares of common stock under the 2023 ATM Program.

Going Concern

Since our inception, we have incurred substantial losses.

As of March 31, 2026, we had an accumulated deficit of $937.5 million and we expect to generate operating losses and negative operating cash flows for the foreseeable

As stated above, as of March 31, 2026, we maintained cash, cash equivalents, and short-term investments of $135.5 million.

As of June 30, 2026, we had an accumulated deficit of $979.6 million and we expect to generate operating losses and negative operating cash flows for the foreseeable future.

As stated above, as of June 30, 2026, we maintained cash, cash equivalents, and short-term investments of $95.1 million.

In accordance with ASC 205-40, we evaluated whether there are conditions and events, considered in the aggregate, that raise substantial doubt about our ability to continue as a going concern within one year after the date on which this Quarterly Report on Form 10-Q is filed.

Based on the our cash, cash equivalents, and short-term investments as of March 31, 2026, our current and forecasted level of operations and forecasted cash flows, our ability to continue as a going concern is dependent upon our ability to obtain the necessary financing to meet our obligations and repay our liabilities arising from normal business operations when they come due.

Based on our cash, cash equivalents, and short-term investments as of June 30, 2026, our current and forecasted level of operations and forecasted cash flows, our ability to continue as a going concern is dependent upon our ability to obtain the necessary financing to meet our obligations and repay our liabilities arising from normal business operations when they come due.

Management plans to provide for capital requirements through financing or other transactions, and selling shares under our “at the market offering” program.

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As a result, we concluded that substantial doubt exists about our ability to continue as a going concern for 12 months from the date these condensed consolidated financial statements are issued.

The condensed consolidated financial statements as of March 31, 2026 have been prepared under the assumption that we will continue as a going concern for the next 12 months and that contemplates the realization of assets and satisfaction of liabilities and commitments in the normal course of business.

Our ability to continue as a going concern is dependent upon our uncertain ability to obtain additional capital, reduce expenditures and/or execute on its business plan.

The condensed consolidated financial statements as of June 30, 2026 have been prepared under the assumption that we will continue as a going concern for the next 12 months and that contemplates the realization of assets and satisfaction of liabilities and commitments in the normal course of business.

Our ability to continue as a going concern is dependent upon our uncertain ability to obtain additional capital, reduce expenditures and/or execute on our business plan.

These condensed consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty.

The following table summarizes our sources and uses of cash for each of the periods presented:

Three Months Ended

Six Months Ended

(in thousands)

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Operating Activities

Net cash used in operating activities for the three months ended March 31, 2026 was driven primarily by the following uses of cash:

Net cash used in operating activities for the six months ended June 30, 2026 was driven primarily by the following uses of cash:

• $91.2 million net loss;

• $4.5 million change in accrued expenses and other current liabilities;

• $3.9 million change in lease liabilities;

• $3.6 million change in accrued expenses and other current liabilities;

• $3.0 million change in accounts payable;

• $2.0 million change in deferred revenue — related party.

These were offset by:

• $18.1 million of non-cash amounts included in net loss, which primarily consisted of stock-based compensation expense, non-cash lease expense, and depreciation expense;

• $3.6 million change in accounts payable;

• $1.9 million change in prepaid expenses and other current assets.

Net cash used in operating activities for the three months ended March 31, 2025 was driven primarily by the following uses of cash:

Net cash used in operating activities for the six months ended June 30, 2025 was driven primarily by the following uses of cash:

• $104.5 million net loss;

• $2.4 million change in deferred revenue — related party;

• $2.1 million change in lease liabilities;

• $1.7 million change in accrued expenses and other current liabilities;

• $1.4 million change in deferred revenue from consideration received from BMS in September 2024.

These were offset by:

• $10.7 million of non-cash amounts included in net loss, which primarily consisted of stock-based compensation expense, non-cash lease expense, depreciation and amortization expense, and change in fair value of short-term investment — related party;

• $1.5 million change in accounts payable.

These were offset by $21.3 million of non-cash amounts included in net loss, which primarily consisted of stock-based compensation expense, non-cash lease expense, depreciation expense, and change in fair value of short-term investment — related party.

Investing Activities

Net cash provided by investing activities for the three months ended March 31, 2026 was driven primarily by $43.6 million of maturities of marketable securities, net of purchases.

Net cash used in investing activities for the three months ended March 31, 2025 was driven primarily by the following:

• $45.3 million of purchases of marketable securities, net of maturities;

Net cash provided by investing activities for the six months ended June 30, 2026 was driven primarily by $67.7 million of maturities of investments, net of purchases.

Net cash used in investing activities for the six months ended June 30, 2025 was driven primarily by the following:

• $41.0 million of purchases of investments, net of maturities;

• $4.0 million of purchases of property and equipment.

Financing Activities

Net cash provided by financing activities for the three months ended March 31, 2025 was driven by $6.0 million of proceeds received under our agreement with Cystic Fibrosis Foundation.

Net cash provided by financing activities for the six months ended June 30, 2025 was driven by $6.0 million of proceeds received under our agreement with Cystic Fibrosis Foundation.

Funding Requirements

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• identify product candidates;

• evaluate strategic alternatives and potential partnership opportunities for PM359, including our ability to execute and realize the anticipated benefits of any strategic alternatives we may pursue;

• develop, maintain, expand and protect our intellectual property portfolio and defend intellectual property-related claims;

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Debt and equity financing, if available, may involve agreements that include restrictive covenants that limit our ability to take specified actions, such as incurring additional debt, making capital expenditures or declaring dividends.

If we raise funds through collaborations, strategic alliances or marketing, or distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, any future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us.

If we raise funds through collaborations, strategic alliances or marketing, or distribution or licensing

arrangements with third parties, we may have to relinquish valuable rights to our technologies, any future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us.

If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, reduce or eliminate our product development or future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

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These contracts generally provide for termination on notice, and therefore are cancellable contracts and not included in the table of contractual obligations and commitments.

During the three months ended March 31, 2026, except for the minimum lease commitments disclosed in Note 6, Leases , to the unaudited condensed consolidated financial statements in this Quarterly Report on Form 10-Q, there were no significant changes to our contractual obligations and commitments described under Management’s Discussion and Analysis of Financial Condition and Results of Operations in our Annual Report on Form 10-K for the year ended December 31, 2025.

During the six months ended June 30, 2026, except for the minimum lease commitments disclosed in Note 6, Leases , to the unaudited condensed consolidated financial statements in this Quarterly Report on Form 10-Q, there were no significant changes to our contractual obligations and commitments described under Management’s Discussion and Analysis of Financial Condition and Results of Operations in our Annual Report on Form 10-K for the year ended December 31, 2025.

Critical Accounting Policies and Significant Judgments and Estimates

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The preparation of these condensed consolidated financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosures of contingent assets and liabilities at the date of the condensed consolidated financial statements and the reported amounts of revenues and expenses incurred during the reporting periods.

judgments about the carrying values of assets and liabilities recorded revenues and expenses that are not readily apparent from other sources.

We base our estimates on historical experience, known trends and events, and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities recorded revenues and expenses that are not readily apparent from other sources.

We evaluate our estimates and assumptions on an ongoing basis.

Actual results may differ from these estimates.

During the three months ended March 31, 2026, there were no material changes to our critical accounting policies and significant judgments described under Management’s Discussion and Analysis of Critical Accounting Policies and Significant Judgments and Estimates which are included in our Annual Report on Form 10-K for the year ended December 31, 2025.

During the six months ended June 30, 2026, there were no material changes to our critical accounting policies and significant judgments described under Management’s Discussion and Analysis of Critical Accounting Policies and Significant Judgments and Estimates which are included in our Annual Report on Form 10-K for the year ended December 31, 2025.

Recently Issued and Adopted Accounting Pronouncements

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As a result of this election, our condensed consolidated financial statements may not be comparable to other public companies that comply with new or revised accounting pronouncements as of public company effective dates.

We may choose to early adopt any new or revised accounting standards whenever such early adoption is permitted for private companies.

We may choose to

early adopt any new or revised accounting standards whenever such early adoption is permitted for private companies.

We are also a “smaller reporting company” meaning that the market value of our stock held by non-affiliates is less than $700 million and our annual revenue was less than $100 million during the most recently completed fiscal year.

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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-Q

Item 1. Financial Statements