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Item 2. Management’s Discussion and Analysis

2023-08-09 compared with 2023-05-05 · 353 added, 327 removed, 381 unchanged (64% of the section changed)

Management’s Discussion and Analysis of Financial Condition and Results of Operations

You should read the following discussion and analysis of our financial condition and results of operations together with the consolidated financial statements and related notes

that are included elsewhere in this Quarterly Report on Form 10-Q and our annual report on Form 10-K, filed on February 28, 2023 with the SEC (the “2022 Form 10-K”).

You should read the following discussion and analysis of our financial condition and results of operations together with the consolidated financial statements and related notes that

are included elsewhere in this Quarterly Report on Form 10-Q and our annual report on Form 10-K, filed on February 28, 2023 with the SEC (the “2022 Form 10-K”).

This discussion contains forward-looking statements based upon current plans,

expectations and beliefs that involve risks and uncertainties.

Our actual results may differ materially from those anticipated in these forward-looking statements as a result of various factors, including, but not limited to, those discussed in

the section entitled “Risk Factors” and elsewhere in this Quarterly Report on Form 10-Q.

In preparing this MD&A, we presume that readers have access to and have read the MD&A in our 2022 Form 10-K, pursuant to Instruction 2 to paragraph

of Item 303 of Regulation S-K.

Unless stated otherwise, references in this Quarterly Report on Form 10-Q to “us,” “we,” “our,” or our “Company” and similar terms refer to Rocket Pharmaceuticals, Inc.

Our actual results may differ materially from those anticipated in these forward-looking statements as a result of various factors, including, but not limited to, those discussed in the

2022 Form 10-K and in this Quarterly Report on Form 10-Q.

In preparing this MD&A, we presume that readers have access to and have read the MD&A in our 2022 Form 10-K.

Unless stated otherwise, references in this Quarterly Report on Form 10-Q

to “us,” “we,” “our,” or our “Company” and similar terms refer to Rocket Pharmaceuticals, Inc.

We are a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best-in-class gene therapies, with direct on-target mechanism of action and

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These include

programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I”), a genetic disorder that causes the

immune system to malfunction and Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia.

Of these, both the Phase 2 FA program and the Phase ½ LAD-I

program produced data read out in 2022 and regulatory filings in the United States (“U.S.”) and Europe (“EU”) are anticipated in 2023.

system to malfunction, and Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia.

Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program

produced data read outs in 2022 and regulatory filings in the United States (“U.S.”) and Europe (“EU”) are anticipated in 2023.

Additional work on a gene therapy program for the less common FA subtypes C and G is ongoing.

In the U.S., we

also have a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure.

The Danon program is currently in an ongoing Phase 1 trial and

pivotal Phase 2 study initiation expected in the second quarter of 2023.

Additionally, we have an AAV vector program targeting Plakophilin-2 Arrhythmogenic Cardiomyopathy (“PKP2-ACM”), an inheritable cardiac disorder that is characterized by a

progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death.

This program, also referred to as Pegasus, will be approaching

IND submission in the second quarter of 2023.

In the U.S., we also

have a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early

death due to heart failure.

The Danon program is currently in an ongoing Phase 1 trial .

Additionally, we have an AAV vector program targeting Plakophilin-2 Arrhythmogenic Cardiomyopathy (“PKP2-ACM”), an inheritable cardiac disorder that is

characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death.

This program, also referred to as Pegasus,

received FDA clearance on an IND and we are in the process of initiating a Phase 1 study.

As a result of our acquisition of Renovacor, Inc.

(“Renovacor”), we are now able to utilize recombinant AAV9-based gene therapy designed to slow or halt progression of

BAG3 Dilated Cardiomyopathy (“DCM”), which is the most common form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that are unable to pump blood.

We have global

commercialization and development rights to all of these product candidates under royalty-bearing license agreements.

(“Renovacor”), we are now able to utilize recombinant AAV9-based gene

therapy designed to slow or halt progression of BAG3 Dilated Cardiomyopathy (“DCM”), which is the most common form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that

are unable to pump blood.

We have global commercialization and development rights to all of these product candidates under royalty-bearing license agreements.

Effective December 2021, a decision was made to no longer pursue Rocket-sponsored clinical evaluation of RP-L401;

this program was returned to academic innovators.

Although we believe that gene

therapy may be beneficial to patients afflicted with this disorder, we have opted to focus available resources towards advancement of RP-A601, RP-A501, RP-L102, RP-L201 RP-L301, and BAG3-DCM based on the compelling clinical data to date and

potential for therapeutic advancement in these severe disorders of childhood and young adulthood.

Although we believe that gene therapy

may be beneficial to patients afflicted with this disorder, we have opted to focus available resources towards advancement of RP-A601, RP-A501, RP-L102, RP-L201 RP-L301, and BAG3-DCM based on the compelling clinical data to date and potential for

therapeutic advancement in these severe disorders of childhood and young adulthood.

Recent Developments

At-the-Market Offering Program

On February 28, 2022, we entered into the Sales Agreement with Cowen with respect to an at-the-market offering program pursuant to which we may offer and sell, from time to time at its sole

discretion, shares through Cowen as our sales agent.

On February 28, 2022, we entered into the Sales Agreement with Cowen with respect to an at-the-market offering program pursuant to which we may offer and sell, from time to time at our sole discretion,

shares through Cowen as our sales agent.

The shares to be offered and sold under the Sales Agreement, if any, will be offered and sold pursuant to our shelf registration statement on Form S-3.

We filed a prospectus supplement with the

SEC on February 28, 2022 in connection with the offer and sale of the shares pursuant to the Sales Agreement.

We filed a prospectus supplement with the SEC on

February 28, 2022 in connection with the offer and sale of the shares pursuant to the Sales Agreement.

We will pay Cowen a cash commission of 3.0% of gross proceeds from the sale of the shares pursuant to the Sales Agreement.

agreed to provide Cowen with customary indemnification and contribution rights.

We also agreed to

provide Cowen with customary indemnification and contribution rights.

We reimbursed Cowen for certain expenses incurred in connection with the Sales Agreement.

Through March 31, 2023, we sold 4.2 million shares under the at-the-market

offering program for gross proceeds of $65.8 million, less commissions of $2.0 million for net proceeds of $63.8 million.

During the three months ended March 31, 2023, the Company sold 0.9 million shares under the at-the-market offering program

for gross proceeds of $17.8 million, less commission of $0.6 million for net proceeds of $17.2 million.

Renovacor Acquisition

On September 19, 2022, the Company entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Renovacor, a Delaware corporation pursuant to which the Company acquired Renovacor (the

Through June 30, 2023, we sold 4.2 million shares under the at-the-market offering

program for gross proceeds of $65.8 million, less commissions of $2.0 million for net proceeds of $63.8 million.

During the six months ended June 30, 2023, we sold 0.9 million shares under the at-the-market offering program for gross proceeds of

$17.8 million, less commission of $0.6 million for net proceeds of $17.2 million.

Renovacor Acquisition

On September 19, 2022, we entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Renovacor, a Delaware corporation pursuant to which we acquired Renovacor (the “Renovacor

Acquisition”).

The Renovacor Acquisition closed on December 1, 2022.

Subject to the terms and conditions of the Merger Agreement, each share of Renovacor’s common stock, par value $0.0001 per share outstanding immediately prior to the

effective time of the Renovacor Acquisition was canceled and converted into the right to receive 0.1763 (the “Exchange Ratio”) fully paid and non-assessable shares of the Company’s common stock, $0.01 par value per share, which was determined on

the basis of an exchange formula set forth in the Merger Agreement.

The Company issued a total of 3,391,976 shares of common stock in connection with the Renovacor Acquisition and incurred approximately $1.3 million of acquisition related costs.

Subject to the terms and conditions of the Merger Agreement, each share of Renovacor’s common stock, par value $0.0001 per share outstanding immediately prior to the effective

time of the Renovacor Acquisition was canceled and converted into the right to receive 0.1763 (the “Exchange Ratio”) fully paid and non-assessable shares of our common stock, which was determined on the basis of an exchange formula set forth in the

Merger Agreement.

We issued a total of 3,391,976 shares of common stock in connection with the Renovacor Acquisition and incurred approximately $1.3 million of acquisition related costs.

Gene Therapy Overview

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We are using modified non-pathogenic viruses for the development of our gene therapy treatments.

Viruses are particularly well suited as delivery vehicles because they are adept at penetrating cells

and delivering genetic material inside a cell.

In creating our viral delivery vehicles, the viral (pathogenic) genes are removed and are replaced with a functional form of the missing or mutant gene that is the cause of the patient’s genetic

The functional form of a missing or mutant gene is called a therapeutic gene, or the “transgene.” The process of inserting the transgene is called “transduction.” Once a virus is modified by replacement of the viral genes with a

transgene, the modified virus is called a “viral vector.” The viral vector delivers the transgene into the targeted tissue or organ (such as the cells inside a patient’s bone marrow).

Viruses are particularly well suited as delivery vehicles because they are adept at penetrating cells and

delivering genetic material inside a cell.

In creating our viral delivery vehicles, the viral (pathogenic) genes are removed and are replaced with a functional form of the missing or mutant gene that is the cause of the patient’s genetic disease.

modified virus is called a “viral vector.” The viral vector delivers the transgene into the targeted tissue or organ (such as the cells inside a patient’s bone marrow).

We have two types of viral vectors in development, LV and AAV.

We believe that our LV and AAV-based programs have the potential to offer a significant therapeutic benefit to patients that is durable (long-lasting).

The gene therapies can be delivered either (1) ex vivo (outside the body), in which case the patient’s cells are extracted and the vector is delivered to

these cells in a controlled, safe laboratory setting, with the modified cells then being reinserted into the patient, or (2) in vivo (inside the body), in which case the vector is injected directly into

the patient, either intravenously (“IV”) or directly into a specific tissue at a targeted site, with the aim of the vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greater regulatory acceptance of gene therapy have created a promising environment to advance gene therapy products as these products

are being designed to restore cell function and improve clinical outcomes, which in many cases include prevention of death at an early age.

The FDA approval of several gene therapies in recent years indicates that there is a regulatory pathway

forward for gene therapy products.

We believe that

our LV and AAV-based programs have the potential to offer a significant therapeutic benefit to patients that is durable (long-lasting).

The gene therapies can be delivered either (1) ex vivo (outside the body), in which case the patient’s cells are extracted and the vector is delivered to these

cells in a controlled, safe laboratory setting, with the modified cells then being reinserted into the patient, or (2) in vivo (inside the body), in which case the vector is injected directly into the

patient, either intravenously (“IV”) or directly into a specific tissue at a targeted site, with the aim of the vector delivering the transgene to the targeted cells.

We believe that scientific advances, clinical progress, and the greater regulatory acceptance of gene therapy have created a promising environment to advance gene therapy products as these products are

being designed to restore cell function and improve clinical outcomes, which in many cases include prevention of death at an early age.

The FDA approval of several gene therapies in recent years indicates that there is a regulatory pathway forward

for gene therapy products.

Pipeline Overview

The chart below shows the current phases of development of Rocket’s programs and product candidates:

The chart below shows the current phases of development of our programs and product candidates:

Cardiovascular Programs

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This mutation results in the accumulation of autophagic vacuoles, predominantly in cardiac and skeletal muscle.

Male patients often require heart transplantation and typically die in their teens or twenties

from progressive heart failure.

Male patients often require heart transplantation and typically die in their teens or twenties from

progressive heart failure.

Along with severe cardiomyopathy, other DD-related manifestations can include skeletal muscle weakness and intellectual impairment.

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associated with significant short- and long-term complications and is not curative of the disorder in the long-term.

RP-A501 is in clinical trials as an in vivo therapy for DD, which is estimated to have

a prevalence of 15,000 to 30,000 patients in the U.S.

RP-A501 is in clinical trials as an in vivo therapy for DD, which is estimated to have a

prevalence of 15,000 to 30,000 patients in the U.S.

DD is an X-linked dominant, monogenic rare inherited disorder characterized by progressive cardiomyopathy which is almost universally fatal in males even in settings where cardiac transplantation is

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We have treated seven patients in the RP-A501 Phase 1 clinical trial, which enrolled adult/older adolescent and pediatric male DD patients.

This includes a first cohort evaluating a low-dose (6.7e13 genome copies (gc)/kilogram (kg)) in adult/older adolescent patients aged 15 or greater (n=3), a second cohort evaluating a higher dose (1.1e14 gc/kg) in adult/older adolescent patients

aged 15 or greater (n=2), and a pediatric cohort at a low dose level (6.7e13 gc/kg;

includes a first cohort evaluating a low-dose (6.7e13 genome copies (gc)/kilogram (kg)) in adult/older adolescent patients aged 15 or greater (n=3), a second cohort evaluating a higher dose (1.1e14 gc/kg) in adult/older adolescent patients aged 15

or greater (n=2), and a pediatric cohort at a low dose level (6.7e13 gc/kg;

As previously disclosed, a patient receiving therapy on the high dose cohort (1.1e14 gc/kg dose) had progressive heart failure and underwent a heart transplant at month five following therapy.

patient had more advanced disease than the four other adult/older adolescent patients who received treatment in the low and high dose cohorts, as evidenced by diminished baseline left ventricle ejection fraction (35%) on echocardiogram and

markedly elevated left ventricle filling pressure prior to treatment.

elevated left ventricle filling pressure prior to treatment.

The patient’s clinical course was characteristic of DD progression.

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Additional safety measures have been implemented and are reflected in the updated trial protocol.

measures include exclusion of patients with end-stage heart failure, and a refined immunomodulatory regimen involving transient B- and T-cell mediated inhibition, with emphasis on preventing complement activation, while also enabling lower

steroid doses and earlier steroid taper, with all immunosuppressive therapy discontinued 2-3 months following administration of RP-A501.

doses and earlier steroid taper, with all immunosuppressive therapy discontinued 2-3 months following administration of RP-A501.

We are conducting a variety of efficacy assessments in the Phase I clinical study to measure the prospect of benefit for patients.

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New York Heart Association (“NYHA”) Functional Classification is the most commonly used heart failure classification system.

NYHA Class II is where a patient exhibits a slight limitation of physical activity, is

comfortable at rest, and ordinary physical activity results in fatigue, palpitation and/or dyspnea.

NYHA Class II is where a patient exhibits a slight limitation of physical activity,

is comfortable at rest, and ordinary physical activity results in fatigue, palpitation and/or dyspnea.

Class I is where a patient exhibits no limitation of physical activity and ordinary physical activity does not cause undue fatigue,

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Brain natriuretic peptide (“BNP”) is a blood-based evaluation and a key marker of heart failure with prognostic significance in CHF and cardiomyopathies.

Elevations in BNP are strongly associated with worsening

heart failure and poor outcomes in cardiovascular disease.

Elevations in BNP are strongly associated with

worsening heart failure and poor outcomes in cardiovascular disease.

High sensitivity troponin I (“hsTnI”) is a blood-based evaluation and a key marker of cardiac injury, one that is (like BNP) frequently elevated in DD patients and has been shown to be markedly elevated in

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considered meaningful and have been shown to correlate with outcomes.

Histologic examination of endomyocardial biopsies via hematoxylin and eosin (“H&E”) histology and electron microscopy is used to detect evidence of DD-associated tissue derangements, including the presence

of autophagic vacuoles and disruption of myofibrillar architecture, each of which are characteristic of DD-related myocardial damage.

LAMP2B gene expression in endomyocardial biopsy samples is measured via both immunohistochemistry and Western blot and confirms the presence of LAMP2B protein in DD cardiac tissue following RP-A501 treatment.

presence of autophagic vacuoles and disruption of myofibrillar architecture, each of which are characteristic of DD-related myocardial damage.

LAMP2B gene expression in endomyocardial biopsy samples is measured via both immunohistochemistry and Western blot and confirms the presence of LAMP2B protein in DD cardiac tissue following RP-A501

In September 2022, we presented interim data for the ongoing Phase 1 trial of RP-A501 at the Heart Failure Society of America (“HFSA”) meeting, including updated safety and initial efficacy parameters

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serious drug product-related adverse events or severe adverse events were observed in both adult/older adolescent and pediatric low dose cohorts.

All observed adverse effects at both doses were reversible and no lasting sequelae were observed

with follow-up of 2-3 years from treatment for the adult/older adolescent cohort and 6-11 months for the pediatric cohort.

Any early transaminase and creatinine kinase elevations returned to baseline or decreased, and any transient exacerbation

of DD-associated skeletal myopathy resolved upon discontinuation of corticosteroid therapy.

The updated safety data presented at HFSA in September 2022 and at AHA in November 2022 reconfirmed that RP-A501 was generally well tolerated at the low

dose with a manageable safety profile across pediatric and adult/older adolescent cohorts.

All observed adverse effects at both doses were reversible and no lasting sequelae were observed with

follow-up of 2-3 years from treatment for the adult/older adolescent cohort and 6-11 months for the pediatric cohort.

Any early transaminase and creatinine kinase elevations returned to baseline or decreased, and any transient exacerbation of

DD-associated skeletal myopathy resolved upon discontinuation of corticosteroid therapy.

The updated safety data presented at HFSA in September 2022 and at AHA in November 2022 reconfirmed that RP-A501 was generally well tolerated at the low dose

with a manageable safety profile across pediatric and adult/older adolescent cohorts.

In the pediatric cohort, an improvement in NYHA Class (from Class II to I) was reported in both patients after 6 and 9 months of follow-up post-RP-A501.

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Improvements (reductions) in hsTnI, a key marker of myocardial injury, were observed in both pediatric patients at 6 and 9 months of follow-up, with levels at these assessments less than 20% of baseline values.

In the adult/older

adolescent cohorts, reductions in hsTnI were observed in three low-dose patients and one high-dose patient, with reductions greater than 50% of baseline levels identified in these four patients on at least one assessment, and reductions sustained

through 24-36 months of follow-up.

In the adult/older adolescent

cohorts, reductions in hsTnI were observed in three low-dose patients and one high-dose patient, with reductions greater than 50% of baseline levels identified in these four patients on at least one assessment, and reductions sustained through

24-36 months of follow-up.

Reductions in BNP of at least 25% below baseline values were identified in three low-dose patients and one high-dose patient on at least one assessment.

In two of the adult/older adolescent patients, BNP levels

were modestly above baseline at the most recent assessment;

In two of the adult/older adolescent patients, BNP levels were

modestly above baseline at the most recent assessment;

however baseline BNP levels were either within normal limits or mildly elevated for these two patients.

In adult/older adolescent cohort patients with closely monitored immunomodulation

(two low-dose and one high-dose) left ventricular posterior wall thickness improved (approximately 15-25% decrease compared to pretreatment baseline) and reductions in left ventricular mass were identified in four patients, including the patient

in the low-dose cohort for whom immunomodulation was not closely monitored.

In adult/older adolescent cohort patients with closely monitored immunomodulation (two

low-dose and one high-dose) left ventricular posterior wall thickness improved (approximately 15-25% decrease compared to pretreatment baseline) and reductions in left ventricular mass were identified in four patients, including the patient in the

low-dose cohort for whom immunomodulation was not closely monitored.

Severe and progressive wall thickening is a hallmark of the hypertrophic cardiomyopathy of Danon disease and is a major contributor to early mortality in male patients.

Evidence of sustained cardiac LAMP2B gene expression by immunohistochemistry with qualitative improvement of vacuoles and cardiac tissue architecture on standard H&E and electron microscopy was observed at both dose levels in four of five

patients in the adult/older adolescent cohorts and both patients in the pediatric cohort.

Sustained cardiac LAMP2B gene expression by immunohistochemistry was observed in all three adult/older adolescent patients with a closely monitored

immunomodulatory regimen through 24 months of follow-up.

Importantly, genetic correction (as evidenced by myocardial vector copy numbers (“VCNs”) and LAMP2 protein expression were accompanied by reductions in the relative area of autophagic

vacuoles relative to overall myocardial area, with decreases in this ratio of at least 20% relative to baseline identified in four adult/older adolescent cohort patients (three of whom had reductions of at least 50%).

Substantial reductions

(>50% baseline) in vacuolar area were also identified in the one pediatric cohort patient for whom this parameter was evaluable at 6 months post-therapy.

In addition to the improvements identified in NYHA Class, improvements in quality of life

(“QOL”) as reported via the KCCQ were noted in three of the adult/older adolescent patients who had closely monitored immunomodulation, and both of the pediatric cohort patients;

KCCQ score at baseline was 50 for the initial pediatric patient and

was 93 at the most recent 9 month assessment;

of sustained cardiac LAMP2B gene expression by immunohistochemistry with qualitative improvement of vacuoles and cardiac tissue architecture on standard H&E and electron microscopy was observed at both dose levels in four of five patients in

the adult/older adolescent cohorts and both patients in the pediatric cohort.

Sustained cardiac LAMP2B gene expression by immunohistochemistry was observed in all three adult/older adolescent patients with a closely monitored immunomodulatory

regimen through 24 months of follow-up.

Importantly, genetic correction (as evidenced by myocardial vector copy numbers (“VCNs”) and LAMP2 protein expression were accompanied by reductions in the relative area of autophagic vacuoles relative to

overall myocardial area, with decreases in this ratio of at least 20% relative to baseline identified in four adult/older adolescent cohort patients (three of whom had reductions of at least 50%).

Substantial reductions (>50% baseline) in

vacuolar area were also identified in the one pediatric cohort patient for whom this parameter was evaluable at 6 months post-therapy.

In addition to the improvements identified in NYHA Class, improvements in quality of life (“QOL”) as reported via

the KCCQ were noted in three of the adult/older adolescent patients who had closely monitored immunomodulation, and both of the pediatric cohort patients;

KCCQ score at baseline was 50 for the initial pediatric patient and was 93 at the most recent

9 month assessment;

KCCQ score at baseline was 52 for the second pediatric patient and was 81 at a preliminary 3 month assessment.

On December 22, 2022, we announced updates from our end-of-Phase 1 meeting with the FDA regarding RP-A501.

During the meeting, we reviewed the positive Phase 1 dataset with the FDA and proposed a

study design and endpoints for ongoing clinical development of the investigational gene therapy.

Following discussions with the FDA, we anticipate proceeding with a dose of 6.7e13 GC/kg, and we anticipate utilizing a single arm open-label trial

design with a robust natural history comparator, pursuant to the FDA’s acknowledgment of the challenges associated with executing a randomized controlled trial in DD.

The FDA has also expressed an openness to considering a biomarker-based

composite endpoint supported by functional and quality-of-life assessments as measures of patient benefit.

We look forward to continued dialogue with the FDA on the design for our proposed pivotal trial, including discussion of appropriate

external controls for the study and appropriate endpoints to support accelerated approval.

We are now in discussion with the FDA about a trial design that will enable evaluation of two pediatric patients treated with drug product manufactured at

our in-house cGMP AAV facility as an initial component of a modestly sized global pivotal study.

During the meeting, we reviewed the positive Phase 1 dataset with the FDA and proposed a study

design and endpoints for ongoing clinical development of the investigational gene therapy.

Following discussions with the FDA, we anticipate proceeding with a dose of 6.7e13 GC/kg, and we anticipate utilizing a single arm open-label trial design

with a robust natural history comparator, pursuant to the FDA’s acknowledgment of the challenges associated with executing a randomized controlled trial in DD.

The FDA has also expressed an openness to considering a biomarker-based composite

endpoint supported by functional and quality-of-life assessments as measures of patient benefit.

We look forward to continued dialogue with the FDA on the design for our proposed pivotal trial, including discussion of appropriate external controls

for the study and appropriate endpoints to support accelerated approval.

We are now in discussion with the FDA about a trial design that will enable evaluation of two pediatric patients treated with drug product manufactured at our in-house cGMP

AAV facility as an initial component of a modestly sized global pivotal study.

On January 9, 2023, we presented additional positive efficacy updates from our Phase I study of RP-A501 during the 41st Annual J.P.

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A summary of these updates is provided in the table below.

We also provided additional natural history comparator

data, which showed the marked divergence of the course of Phase I patients from that of untreated patients in terms of key biomarkers (BNP) and functional measures (NYHA Class).

Furthermore, RP-A501 continued to be well tolerated at 2-3 years

post treatment in both adult/older adolescent high and low-dose cohorts and at 8 to 13 months in the pediatric cohort.

In the pediatric cohort, no significant immediate or delayed toxicities, significant skeletal myopathy, or late transaminase

elevation have been observed.

We also provided additional natural history comparator data,

which showed the marked divergence of the course of Phase I patients from that of untreated patients in terms of key biomarkers (BNP) and functional measures (NYHA Class).

Furthermore, RP-A501 continued to be well tolerated at 2-3 years post

treatment in both adult/older adolescent high and low-dose cohorts and at 8 to 13 months in the pediatric cohort.

In the pediatric cohort, no significant immediate or delayed toxicities, significant skeletal myopathy, or late transaminase elevation

have been observed.

Improvement or Stabilization Observed Across Key Biomarker, Echo Findings and Functional Measures in Phase 1 RP-A501 study

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In addition to these clinical updates, we also provided updates on our in-house manufacturing activities.

We have successfully produced 2 cGMP RP-A501 batches that have superior specifications to

Phase I material in both titer and full versus empty particles.

We have successfully produced 2 cGMP RP-A501 batches that have superior specifications to Phase

I material in both titer and full versus empty particles.

We believe the improved quality of our in-house manufactured product will allow for full dosing with lower total viral particles, potentially further optimizing the safety profile of

RP-A501.Furthermore, we have agreement from the FDA on the continued utilization of HEK-293 cell-based process through commercialization as well as our comparability approach and potency assay.

In May 2023 we presented previously disclosed results from the Phase I study of RP-A501 at the ASGCT 26th Annual Meeting, As of the most recent date extraction, all six patients that remain in follow-up

continued to show signs of improvement or stabilization.

Results from the ongoing Phase 1 DD trial represent one of the most comprehensive investigational gene therapy datasets for any cardiac condition.

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Anticipated Milestones

On February 7, 2023, we announced that RP-A501 received RMAT designation from the FDA.

We are very encouraged by the highly collaborative ongoing dialogue with the FDA for RP-A501 in DD and subject to

the continued dialogue and agreement with the FDA anticipate initiating the initial component of the global study in the second quarter of 2023.

On February 7, 2023, we announced that RP-A501 received RMAT designation from the FDA, and on May 31, 2023, we received PRIME designation from the EMA.

We are very encouraged by the highly collaborative

ongoing dialogue with the FDA for RP-A501 in DD and are prepared to initiate the global study pending agreement with the Agency on the key components of the proposed study design.

Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

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However, since left dominant and biventricular forms have also been observed, this has led more recently to the use of the term ACM.

Mutations in the PKP2 gene comprise the most frequent genetically

identified etiology of familial ACM.

Mutations in the PKP2 gene comprise the most frequent genetically identified

etiology of familial ACM.

PKP2 encodes for the protein Plakophilin-2, which is a component of the desmosome, an intercellular complex involved in cell-cell adhesion.

PKP2 is also involved in transcriptional regulation of calcium

signaling between cardiomyocytes.

PKP2 is also involved in transcriptional regulation of calcium signaling between

cardiomyocytes.

Patients with mutations in PKP2 are typically heterozygous and demonstrate reduced expression of PKP2 in the myocardium.

Mean presentation is at the age of 35, and patients have a very high lifetime risk of

ventricular arrhythmias, structural ventricular abnormalities, and sudden cardiac death (“SCD”).

There are no specific available medical therapies available that have been shown to be highly effective for ACM, and current treatment protocols follow standard ventricular arrhythmia and

cardiomyopathy guidelines, which involve lifestyle modifications (i.e.

Mean presentation is at the age of 35, and patients have a very high lifetime risk of ventricular arrhythmias,

structural ventricular abnormalities, and sudden cardiac death (“SCD”).

There are no specific available medical therapies available that have been shown to be highly effective for ACM, and current treatment protocols follow standard ventricular arrhythmia and cardiomyopathy

guidelines, which involve lifestyle modifications (i.e.

exercise limitation) and include drug treatments such as beta blockers, anti-arrhythmics and diuretics.

The use of these therapies is driven by the arrhythmia burden and

severity of cardiomyopathy.

The use of these therapies is driven by the arrhythmia burden and severity of

cardiomyopathy.

These therapies do not modify the course of the disease, and generally provide only symptomatic and/or palliative support.

Upon diagnosis, a substantial percentage of patients receive an implantable cardiac

defibrillator (“ICD”) for primary or secondary prevention of ventricular arrhythmias and SCD.

Upon diagnosis, a substantial percentage of patients receive an implantable cardiac defibrillator (“ICD”) for

primary or secondary prevention of ventricular arrhythmias and SCD.

Of note, ICDs are not curative, and breakthrough life-threatening arrythmias may persist with ongoing risk of death;

ICDs furthermore do not prevent the

progression to end-stage heart failure.

ICDs furthermore do not prevent the progression to end-stage

heart failure.

ICD firings, although lifesaving, are physically and emotionally traumatic events.

Patients whose condition progresses to end-stage heart failure are considered for cardiac transplantation which, while

curative of underlying disease, is itself associated with significant morbidity and mortality.

Patients whose condition progresses to end-stage heart failure are considered for cardiac transplantation which, while curative of underlying disease,

is itself associated with significant morbidity and mortality.

Hence there exists a high unmet medical need in this population.

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We currently have one adeno-associated viral vector program targeting PKP2-ACM, RP-A601, which is a recombinant AAVrh.74 vector expressing PKP2a.

PKP2-ACM is typically caused by heterozygous

pathogenic mutations in the PKP2 gene resulting in reduced PKP2 expression in the myocardium.

A once-administered gene therapy that addresses the root cause of the disease (PKP2 deficiency) early in the disease course, could mitigate the early

electrical remodeling and diminish the risk of life-threatening arrhythmias and SCD associated with ACM, potentially impeding the development of irreversible cardiac structural changes.

Prevention of syncopal episodes, life-threatening

arrythmias, SCD, ICD shocks and the resulting anxiety, discomfort and hospitalizations is anticipated to result in a vastly improved quality of life and survival benefit.

Furthermore, such an approach could spare patients the need for lifelong

adherence to multiple arrhythmia and heart failure drugs that are nonspecific for PKP2-ACM and are associated with their own side effects, enabling patients an opportunity to live without exercise restrictions and with diminished concern for

arrhythmias, palpitations, ICD shocks and progression to end-stage heart failure.

In nonclinical studies conducted by the Sponsor, RP-A601 has demonstrated efficacy in altering the natural history of PKP2-driven ACM.

PKP2 cKO animals treated with the study drug have exhibited

extended survival to the longest timepoint measured (5 months), reduced cardiac dilation and fibrofatty replacement / fibrosis of the myocardium, preserved left ventricular function, and mitigation of the arrhythmic phenotype.

Untreated PKP2 cKO

mice had a median survival of approximately one month.

PKP2-ACM is typically caused by heterozygous pathogenic

mutations in the PKP2 gene resulting in reduced PKP2 expression in the myocardium.

A once-administered gene therapy that addresses the root cause of the disease (PKP2 deficiency) early in the disease course, could mitigate the early electrical

remodeling and diminish the risk of life-threatening arrhythmias and SCD associated with ACM, potentially impeding the development of irreversible cardiac structural changes.

Prevention of syncopal episodes, life-threatening arrythmias, SCD, ICD

shocks and the resulting anxiety, discomfort and hospitalizations is anticipated to result in a vastly improved quality of life and survival benefit.

Furthermore, such an approach could spare patients the need for lifelong adherence to multiple

arrhythmia and heart failure drugs that are nonspecific for PKP2-ACM and are associated with their own side effects, enabling patients an opportunity to live without exercise restrictions and with diminished concern for arrhythmias, palpitations,

ICD shocks and progression to end-stage heart failure.

In May 2023 we presented preclinical efficacy date for RP-A601 at the American Society of Gene and Cell Therapy 26 t h Annual meeting.

Nonclinical studies conducted by the Sponsor, RP-A601 have demonstrated efficacy

in altering the natural history of PKP2-driven ACM.

100% of PKP2 cKO animals treated with the study drug exhibited extended survival to the longest timepoint measured (5 months), reduced cardiac dilation and fibrofatty replacement / fibrosis of

the myocardium, preserved left ventricular function, and mitigation of the arrhythmic phenotype.

Untreated PKP2 cKO mice had a median survival of approximately one month.

Anticipated Milestones

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appropriate potency assay to support a Phase I study.

We anticipate filing an IND in the second quarter of 2023.

On May 9, 2023, we announced FDA clearance of the IND and on June 8, 2023 we announced receipt of FDA Fast Track and Orphan Drug Designations.

We are in the process of initiating the Phase 1

BAG3 Dilated Cardiomyopathy

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heart transplant, or have heart failure related death at 12 months after diagnosis, nearly twice the rate of similarly staged non-BAG3DCM patients.

In December 2022 we completed our acquisition of Renovacor which provided Rocket with Renovacor’s most advanced program, a recombinant AAV9-based gene therapy designed to deliver a fully functional

BAG3 gene to augment BAG3 protein levels in cardiomyocytes and slow or halt progression of BAG3-DCM.

Initial proof of concept for AAV9-BAG3 has been demonstrated in studies of BAG3-knockout mouse models, which show treated mice have improved

ejection fraction versus untreated knockout mice and comparable ejection fraction to walk test controls at timepoints 4- and 6-weeks post injection.

In December 2022 we completed our acquisition of Renovacor which provided Rocket with Renovacor’s most advanced program, a recombinant AAV9-based gene therapy designed to deliver a fully functional BAG3

gene to augment BAG3 protein levels in cardiomyocytes and slow or halt progression of BAG3-DCM.

Initial proof of concept for AAV9-BAG3 has been demonstrated in studies of BAG3-knockout mouse models, which show treated mice have improved ejection

fraction versus untreated knockout mice and comparable ejection fraction to walk test controls at timepoints 4- and 6-weeks post injection.

Anticipated Milestones

We are in the process of evaluating the optimal development pathway for this program and plan to submit an IND for BAG3-DCM in the first half of 2024.

We are in the process of evaluating the optimal development pathway for this program and plan to submit an IND for BAG3-DCM in 2024.

Hematology Programs

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FA, a rare and life-threatening DNA-repair disorder, generally arises from a mutation in a single FA gene.

An estimated 60 to 70% of cases arise from mutations in the Fanconi-A (“FANCA”) gene, which

is the focus of our program.

An estimated 60 to 70% of cases arise from mutations in the Fanconi-A (“FANCA”) gene, which is

the focus of our program.

FA results in bone marrow failure, developmental abnormalities, myeloid leukemia, and other malignancies, often during the early years and decades of life.

Bone marrow aplasia, which is bone marrow that no longer

produces any or very few red and white blood cells and platelets leading to infections and bleeding, is the most frequent cause of early morbidity and mortality in FA, with a median onset before 10 years of age.

Leukemia is the next most common

cause of mortality, ultimately occurring in about 20% of patients later in life.

Bone marrow aplasia, which is bone marrow that no longer produces

any or very few red and white blood cells and platelets leading to infections and bleeding, is the most frequent cause of early morbidity and mortality in FA, with a median onset before 10 years of age.

Leukemia is the next most common cause of

mortality, ultimately occurring in about 20% of patients later in life.

Solid organ malignancies, such as head and neck cancers, can also occur, although at lower rates during the first two to three decades of life.

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in the GI tract, liver toxicity and skin rashes, as well as increased risk of subsequent cancers.

Our gene therapy program in FA is designed to enable a minimally toxic hematologic correction using a patient’s own stem cells during the early

years of life.

Our gene therapy program in FA is designed to enable a minimally toxic hematologic correction using a patient’s own stem cells during the early years

We believe that the development of a broadly applicable autologous gene therapy can be transformative for these patients.

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The prevalence of FA in the U.S.

and EU is estimated to be approximately 4,000

patients in total.

and EU is estimated to be approximately 4,000 patients

In light of the efficacy seen in non-conditioned patients, the addressable annual market opportunity is now believed to be 400 to 500 patients collectively in the U.S.

We currently have one ex-vivo LV-based program targeting FA, RP-L102.

RP-L102 is our lead LV-based program that we in-licensed from Centro de Investigaciones Energéticas, Medioambientales y

Tecnológicas (“CIEMAT”), which is a leading research institute in Madrid, Spain.

RP-L102 is currently being studied in our Phase 2 registrational enabling clinical trials treating FA patients at the Center for Definitive and Curative Medicine at

Stanford University School of Medicine (“Stanford”), the University of Minnesota, Great Ormond Street Hospital (“GOSH”) in London and Hospital Infantil de Nino Jesus (“HNJ”) in Spain.

RP-L102 is our lead LV-based program that we in-licensed from Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas

(“CIEMAT”), which is a leading research institute in Madrid, Spain.

RP-L102 is currently being studied in our Phase 2 registrational enabling clinical trials treating FA patients at the Center for Definitive and Curative Medicine at Stanford

University School of Medicine (“Stanford”), the University of Minnesota, Great Ormond Street Hospital (“GOSH”) in London and Hospital Infantil de Nino Jesus (“HNJ”) in Spain.

The trial has enrolled a total of ten patients from the U.S.

Two additional patients were treated in the US Phase 1 study at Stanford such that a total of 12 patients have received RP-L102 on Rocket-sponsored clinical trials.

Patients receive a single intravenous infusion of RP-L102 that utilizes

fresh cells and “Process B” which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector and final drug product.

additional patients were treated in the US Phase 1 study at Stanford such that a total of 12 patients have received RP-L102 on Rocket-sponsored clinical trials.

Patients receive a single intravenous infusion of RP-L102 that utilizes fresh cells and

“Process B” which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector and final drug product.

Resistance to mitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum time point of one year post treatment is the primary endpoint for our ongoing Phase 2 study.

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the FDA and EMA, engraftment leading to bone marrow restoration exceeding a 10% mitomycin-C resistance threshold could support a marketing application for approval.

In May 2022, we presented topline data for RP-L102 at ASGCT’s 25 th Annual Meeting.

Five of nine evaluable patients as

of the April 4, 2022 cut-off date had increased resistance to MMC in bone marrow-derived colony forming cells, ranging from 21% to 42% at 12 to 18 months, increasing to 51% to 94% at 18 – 21 months.

The primary endpoint has been achieved, based

on a trial protocol in which statistical and clinical significance requires a minimum of five patients to attain increased MMC resistance at least 10% above baseline at two or more timepoints, and concomitant evidence of genetic correction and

clinical stabilization.

A sixth patient has displayed evidence of progressively increasing genetic correction as evidenced by peripheral VCN.

Three additional patients were less than 12 months post-treatment at the time of presentation.

patient had progressive bone marrow failure following therapy and underwent successful allogeneic transplant as previously disclosed.

The tolerability profile of RP-L102 appears favorable with no signs of dysplasia, clonal dominance or oncogenic

integrations;

as previously reported, one patient experienced a Grade 2 transient infusion-related reaction, which resolved.

In October 2022, we presented data for RP-L102 at the European Society for Cell and Gene Therapy 29 th Annual Meeting,

including the clinical activity results presented at the ASGCT 2022 meeting.

We also disclosed at least one of the additional three patients in our Phase 2 trial of RP-L102 for FA for whom there is less than 12 months of follow-up has

demonstrated initial evidence of engraftment (as demonstrated by bone marrow mitomycin-C resistance and VCN in blood and bone marrow) at levels comparable to those seen in the five patients for whom there is longer-term evidence of progressive

engraftment and phenotypic correction.

We also disclosed at least one of the additional three patients in our Phase 2 trial of RP-L102 for FA for whom there is less than 12 months of follow-up has demonstrated

initial evidence of engraftment (as demonstrated by bone marrow mitomycin-C resistance and VCN in blood and bone marrow) at levels comparable to those seen in the five patients for whom there is longer-term evidence of progressive engraftment and

phenotypic correction.

We also disclosed that one of the initial five patients in this trial who had evidence of engraftment developed a T-cell lymphoblastic lymphoma approximately 22 months after RP-L102 administration.

surgical biopsy of the lymphoma indicated negligible gene markings (VCN of 0.003) at a juncture when concomitant VCN in blood and bone marrow were 0.26 and 0.42 respectively.

These findings conclusively indicate that the lymphoma did not result

from a LV-mediated insertion, as there were essentially no gene markings in the tumor (the very low but detectable VCN is likely the result of blood cells in the tumor specimen).

FA is a cancer-predisposition syndrome and cancers may develop in

patients under the age of 10.

A surgical biopsy of the

lymphoma indicated negligible gene markings (VCN of 0.003) at a juncture when concomitant VCN in blood and bone marrow were 0.26 and 0.42 respectively.

These findings conclusively indicate that the lymphoma did not result from a LV-mediated

insertion, as there were essentially no gene markings in the tumor (the very low but detectable VCN is likely the result of blood cells in the tumor specimen).

FA is a cancer-predisposition syndrome and cancers may develop in patients under the age

Importantly, the patient tolerated induction chemotherapy for the lymphoma without significant complications and is currently in a complete response.

The presence of gene-corrected hematopoietic cells may have

contributed to this patient’s overall tolerance of chemotherapy.

The presence of gene-corrected hematopoietic cells may have contributed to this patient’s

overall tolerance of chemotherapy.

In December 2022, we presented positive clinical data for RP-L102 at the 64 th Annual Meeting of ASH.

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phenotypic correction in at least six of 10 evaluable patients with ≥12 months of follow-up as demonstrated by increased resistance to MMC in bone marrow derived colony forming cells, concomitant genetic correction and hematologic stabilization.

A seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and bone marrow VCN’s, with recent development of MMC resistance and possible indicators of hematologic stability after 36

seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and bone marrow VCN’s, with recent development of MMC resistance and possible indicators of hematologic stability after 36

months of follow-up.

The primary endpoint has been achieved, based on a trial protocol in which statistical and clinical significance requires a minimum of five patients to attain increased MMC resistance at least 10% above baseline at two or

more timepoints and concomitant evidence of genetic correction and clinical stabilization.

timepoints and concomitant evidence of genetic correction and clinical stabilization.

The safety profile of RP-L102 has been highly favorable, and the treatment, administered without any cytotoxic conditioning, has been well tolerated.

signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102 have been observed.

bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102 have been observed.

In May 2023 we presented updated clinical data for RP-L102 at the ASGCT 26th Annual Meeting.

As of the data cut-off (April 17, 2023), RP-L102 conferred sustained genetic correction in eight of 12

evaluable patients and comprehensive phenotypic correction in seven of 12 evaluable patients with ≥12 months of follow up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells and hematologic

stabilization.

The safety profile of RP-L102 continues to be highly favorable with no signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102.

Polyclonal integration patterns have been observed in each of the

seven patients with phenotypic, genetic, and hematologic evidence of engraftment.

Pivotal trial enrollment and treatment have been completed.

Anticipated Milestones

Based on achievement of the primary endpoint as defined in our pivotal Phase 2 study for FA, we have initiated FDA dialogue around biologics license application (“BLA”) filing plans for RP-L102 for

the treatment of FA and anticipate making such filing in the fourth quarter of 2023.

treatment of FA and anticipate making such filing in the fourth quarter of 2023.

Leukocyte Adhesion Deficiency-I (LAD-I)

LAD-I is a rare autosomal recessive disorder of white blood cell adhesion and migration, resulting from mutations in the ITGB2 gene encoding for the Beta-2 Integrin component, CD18.

Deficiencies in

CD18 result in an impaired ability for neutrophils (a subset of infection-fighting white blood cells) to leave blood vessels and enter tissues where these cells are needed to combat infections.

As is the case with many rare diseases, accurate

estimates of incidence are difficult to confirm;

Deficiencies in CD18

result in an impaired ability for neutrophils (a subset of infection-fighting white blood cells) to leave blood vessels and enter tissues where these cells are needed to combat infections.

As is the case with many rare diseases, accurate estimates

of incidence are difficult to confirm;

however, several hundred cases have been reported to date.

Most LAD-I patients are believed to have the severe form of the disease.

Severe LAD-I is notable for recurrent, life-threatening

infections and substantial infant mortality in patients who do not receive an allogeneic HSCT.

Severe LAD-I is notable for recurrent, life-threatening infections and

substantial infant mortality in patients who do not receive an allogeneic HSCT.

Mortality for severe LAD-I has been reported as 60 to 75% by age two in the absence of allogeneic HCST.

We currently have one ex - vivo program targeting LAD-I, RP-L201.

RP-L201 is a clinical program that we in-licensed

RP-L201 is a clinical program that we in-licensed from

We have partnered with UCLA to lead U.S.

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UCLA and its Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research is serving as the lead U.S.

clinical research

center for the registrational clinical trial for LAD-I, and HNJ and GOSH serving as the lead clinical sites in Spain and London, respectively.

This study has received a $6.6 million CLIN2 grant award from the California Institute for Regenerative

Medicine (“CIRM”) to support the clinical development of gene therapy for LAD-I.

The open-label, single-arm, Phase 1/2 registration-enabling clinical trial of RP-L201 has treated nine severe LAD-I patients to assess the safety and tolerability of RP-L201 to

The first patient was treated at UCLA with RP-L201 in the third quarter of 2019.

clinical research center for

the registrational clinical trial for LAD-I, and HNJ and GOSH serving as the lead clinical sites in Spain and London, respectively.

This study has received a $6.6 million CLIN2 grant award from the California Institute for Regenerative Medicine

(“CIRM”) to support the clinical development of gene therapy for LAD-I.

The open-label, single-arm, Phase 1/2 registration-enabling clinical trial of RP-L201 has treated nine severe LAD-I patients to assess the safety and tolerability of RP-L201 to date.

The first patient

was treated at UCLA with RP-L201 in the third quarter of 2019.

Enrollment is now complete in both the Phase 1 and 2 portions of the study;

nine patients have received RP-L201 at 3 investigative centers in the U.S.

In December 2021, we presented positive clinical data at the 63 rd Annual Meeting of ASH.

The ASH oral presentation

included preliminary data from eight of nine severe LAD-I patients, as defined by CD18 expression of less than 2%, who received RP-L201 treatment as of the November 8, 2021, data cut-off date.

Eight patients had follow-up data of at least three

months, and four of the eight patients had been followed for 12 months or longer.

All infusions of RP-L201 were well tolerated and no drug product-related serious adverse events were reported.

Evidence of preliminary efficacy was observed in all

eight evaluable patients.

All eight patients demonstrated neutrophil CD18 expression that exceeded the 4-10% threshold associated with survival into adulthood and consistent with reversal of the severe LAD-I phenotype including six patients with

at least 6 months of follow-up.

Peripheral blood VCN levels have been stable and in the 0.54 – 2.94 copies per genome range.

No patients had LAD-I related infections requiring hospitalization after hematopoietic reconstitution post-RP-L201.

Additional updates presented in January 2022 included a ninth patient achieving CD18 expression of 61% at 3 months, with the preliminary observation that all nine of nine patients have demonstrated 26% to 87% CD18 expression at timepoints ranging

from 3 to 24 months following RP-L102, with stable CD18 expression levels for each patient subsequent to month 3.

In May 2022, we presented updated data at ASGCT’s 25th Annual Meeting.

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patients and overall survival data, including survival data for the seven patients with at least 12 months of follow-up after infusion as of the March 9, 2022 cut-off date.

All patients, aged three months to nine years, demonstrated sustained

CD18 restoration and expression on more than 10% of neutrophils (range:

All patients, aged three months to nine years, demonstrated sustained CD18

restoration and expression on more than 10% of neutrophils (range:

20%-87%, median:

At one year, the overall survival without allogeneic hematopoietic stem cell transplantation across the cohort is 100% based on the Kaplan-Meier

As of the data cut-off, all nine patients are alive and clinically stable.

At one year, the overall survival without allogeneic hematopoietic stem cell transplantation across the cohort is 100% based on the Kaplan-Meier estimate.

of the data cut-off, all nine patients are alive and clinically stable.

All patients demonstrated a statistically significant reduction in the rate of all-cause hospitalizations and severe infections, relative to pre-treatment.

Evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities has been shown along with sustained phenotypic correction.

The tolerability profile of RP-L201 has been highly favorable in all patients with no

RP-L201-related adverse events.

resolution of LAD-I-related skin rash and restoration of wound repair capabilities has been shown along with sustained phenotypic correction.

The tolerability profile of RP-L201 has been highly favorable in all patients with no RP-L201-related

adverse events.

Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and consistent with the tolerability profiles of those agents and procedures.

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events to date.

In May 2023 at the ASGCT 26 th annual meeting, we presented updated top-line data at 12 to 24 months of follow-up for all

nine patients showing 100% overall survival at 12 months post-infusion.

All patients continue to demonstrate evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities, and the safety profile of RP-L201 remains

highly favorable with follow-up of 12-36 months.

No evidence of replication-competent lentivirus has been observed.

Insertion site analyses indicate highly polyclonal integration patterns across the entire cohort.

Anticipated Milestones

Based on the positive efficacy and safety data from the Phase 2 pivotal LAD-I trial, we have initiated discussions with the FDA on BLA filing plans for RP-L201 for the treatment of severe LAD-I and

anticipate making such filing in the second quarter of 2023.

BLA filing submitted to FDA for RP-L201 with approval expected in the second half of 2024.

Pyruvate Kinase Deficiency (PKD)

Red blood cell PKD is a rare autosomal recessive disorder resulting from mutations in the pyruvate kinase L/R (“PKLR”) gene encoding for a component of the red blood cell (“RBC”) glycolytic pathway.

PKD is characterized by chronic non-spherocytic hemolytic anemia, a disorder in which RBCs do not assume a normal spherical shape and are broken down, leading to decreased ability to carry oxygen to cells, with anemia severity that can range from

mild (asymptomatic) to severe forms that may result in childhood mortality or a requirement for frequent, lifelong RBC transfusions.

The pediatric population is the most commonly and severely affected subgroup of patients with PKD, and PKD often

results in splenomegaly (abnormal enlargement of the spleen), jaundice and chronic iron overload which is likely the result of both chronic hemolysis and the RBC transfusions used to treat the disease.

The variability in anemia severity is

believed to arise in part from the large number of diverse mutations that may affect the PKLR gene.

is characterized by chronic non-spherocytic hemolytic anemia, a disorder in which RBCs do not assume a normal spherical shape and are broken down, leading to decreased ability to carry oxygen to cells, with anemia severity that can range from mild

(asymptomatic) to severe forms that may result in childhood mortality or a requirement for frequent, lifelong RBC transfusions.

The pediatric population is the most commonly and severely affected subgroup of patients with PKD, and PKD often results

in splenomegaly (abnormal enlargement of the spleen), jaundice and chronic iron overload which is likely the result of both chronic hemolysis and the RBC transfusions used to treat the disease.

The variability in anemia severity is believed to

arise in part from the large number of diverse mutations that may affect the PKLR gene.

Estimates of disease incidence have ranged between 3.2 and 51 cases per million in the white U.S.

and EU population.

Industry estimates suggest at

least 2,500 cases in the U.S.

Industry estimates suggest at least 2,500

cases in the U.S.

and EU have already been diagnosed despite the lack of FDA-approved molecularly targeted therapies.

Market research indicates the application of gene therapy to broader populations could increase the market

opportunity from approximately 250 to 500 patients per year.

Market research indicates the application of gene therapy to broader populations could increase the market opportunity from

approximately 250 to 500 patients per year.

We currently have one ex - vivo LV-based program targeting PKD, RP-L301.

RP-L301 is a clinical

stage program that we in-licensed from CIEMAT.

RP-L301 is a clinical stage

program that we in-licensed from CIEMAT.

The IND for RP-L301 to initiate the global Phase 1 study cleared in October 2019.

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assess RP-L301 in pediatric (age 8-17) and adult populations.

The trial is designed to assess the safety, tolerability, and preliminary activity of RP-L301, and initial safety evaluation will occur in the adult cohort before evaluation in

pediatric patients.

The trial is designed to assess the safety, tolerability, and preliminary activity of RP-L301, and initial safety evaluation will occur in the adult cohort before evaluation in pediatric

Stanford will serve as the lead site in the U.S.

for adult and pediatric patients, HNJ will serve as the lead site in Europe for pediatrics, and Hospital Universitario Fundación Jiménez Díaz will serve as the lead site in

Europe for adult patients.

for adult and pediatric patients, HNJ will serve as the lead site in Europe for pediatrics, and Hospital Universitario Fundación Jiménez Díaz will serve as the lead site in Europe for adult

In July 2020, we treated the first patient in our clinical trial of RP-L301.

In May 2022, we presented updated data at the 25 th Annual Meeting of the ASGCT.

The presentation included data from two

adult patients with severe or transfusion-dependent anemia as of the April 13, 2022 cut-off date.

At 18 months post-infusion, both patients had sustained transgene expression, normalized hemoglobin, improved hemolysis, no red blood cell

transfusion requirements post-engraftment and improved QOL both reported anecdotally and as documented via formal QOL assessments.

The tolerability profile of RP-L301 appears favorable, with no RP-L301-related serious adverse events through 18

months post-infusion.

Transient transaminase elevation was seen in both patients post-therapy/conditioning, with no clinical stigmata of liver injury and subsequent resolution without clinical sequelae.

In December 2022, we presented positive clinical data at the 64 th Annual Meeting of ASH.

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and there has been no evidence of insertional mutagenesis.

In May 2023, we presented positive updated clinical data at the ASGCT 26th Annual Meeting (data cut-off May 3, 2023), which included up to 30 months of follow-up from the two treated adult patients and

early clinical data from the first pediatric patient treated with RP-L301.

Robust and sustained efficacy was observed in both adult patients at up to 30 months post-infusion evidenced by normalized hemoglobin (from baseline pre-treatment levels in

the 7.0-7.5 g/dL range), improved hemolysis parameters, and red blood cell transfusion independence.

Furthermore, both adult patients reported improved quality of life with documented improvements via formal quality of life assessments.

profile continues to appear highly favorable, with no RP-L301-related serious adverse events in either of the adult patients.

Insertion site analyses in peripheral blood and bone marrow in both adult patients through 24 months post-RP-L301

demonstrated highly polyclonal patterns and there has been no evidence of insertional mutagenesis.

Early results from the first pediatric patient show preliminary safety and efficacy in the adult cohort.

The first pediatric patient infusion of

RP-L301 was well tolerated, with engraftment achieved at day +15, hospital discharge less than one month following infusion, no RP-L301related serious adverse events and early signs of efficacy.

There were no red blood cell transfusion requirements

following engraftment.

Both adult and pediatric enrollment is completed in the Phase 1 study.

Anticipated Milestones

Enrollment in the PKD adult and pediatric cohort is completed in the Phase 1 study.

On May 23, 2023, we announced receipt of FDA RMAT designation for PR-L301 based on the robust

efficacy observed in the Phase 1 treated patients.

In July 2023 we received PRIME designation from EMA.

Initiation of the phase 2 pivotal trial is anticipated in the fourth quarter of 2023.

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Chief Technical Officer.

We seek to bring hope and relief to patients with devastating, undertreated, rare pediatric diseases through the development and commercialization of potentially curative first-in-class gene

We seek to bring hope and relief to patients with devastating, undertreated, rare pediatric diseases through the development and commercialization of potentially curative first-in-class gene therapies.

To achieve these objectives, we intend to develop into a fully-integrated biotechnology company.

In the near and medium-term, we intend to develop our first-in-class product candidates, which are targeting devastating diseases with

substantial unmet need, develop proprietary in-house analytics and manufacturing capabilities and continue to commence registration trials for our currently planned programs.

In the medium and long-term, pending favorable data, we expect to

submit BLAs for the rest of our suite of clinical programs, and establish our gene therapy platform and expand our pipeline to target additional indications that we believe to be potentially compatible with our gene therapy technologies.

addition, during that time, we believe that our currently planned programs will become eligible for priority review vouchers from the FDA that provide for expedited review.

We have assembled a leadership and research team with expertise in cell

and gene therapy, rare disease drug development and product approval.

In the near and medium-term, we intend to develop our first-in-class product candidates, which are targeting devastating diseases with substantial

unmet need, develop proprietary in-house analytics and manufacturing capabilities and continue to commence registration trials for our currently planned programs.

In the medium and long-term, pending favorable data, we expect to submit BLAs for the

rest of our suite of clinical programs, and establish our gene therapy platform and expand our pipeline to target additional indications that we believe to be potentially compatible with our gene therapy technologies.

In addition, during that time,

we believe that our currently planned programs will become eligible for priority review vouchers from the FDA that provide for expedited review.

We have assembled a leadership and research team with expertise in cell and gene therapy, rare disease

drug development and product approval.

We believe that our competitive advantage lies in our disease-based selection approach, a rigorous process with defined criteria to identify target diseases.

We believe that this

approach to asset development differentiates us as a gene therapy company and potentially provides us with a first-mover advantage.

We believe that this approach to asset

development differentiates us as a gene therapy company and potentially provides us with a first-mover advantage.

Financial Overview

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product sales.

From inception through March 31, 2023, we raised net cash proceeds of approximately $852.8 million from investors through both equity and convertible debt financing to fund operating activities.

From inception through June 30, 2023, we raised net cash proceeds of approximately $852.8 million from investors through both equity and convertible debt financing to fund operating activities.

To date, we have not generated any revenue from any sources, including from product sales, and we do not expect to generate any revenue from the sale of products in the near future.

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Our direct R&D expenses by program also include fees incurred under license agreements.

Our personnel, non-program

and unallocated program expenses include costs associated with activities performed by our internal R&D organization and generally benefit multiple programs.

These costs are not separately allocated by product candidate and consist primarily

Our personnel, non-program and

unallocated program expenses include costs associated with activities performed by our internal R&D organization and generally benefit multiple programs.

These costs are not separately allocated by product candidate and consist primarily of:

salaries and personnel-related costs, including benefits, travel, and stock-based compensation, for our scientific personnel performing R&D activities;

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R&D expenses.

The following table presents R&D expenses tracked on a program-by-program basis as well as by type and nature of expense for the three months ended March 31, 2023 and 2022.

Three Months Ended March 31,

The following table presents R&D expenses tracked on a program-by-program basis as well as by type and nature of expense for the three and six months June 30, 2023 and 2022.

Three Months Ended June 30,

Six Months Ended June 30,

Direct Expenses:

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the timing and receipt of any regulatory approvals.

We expect R&D expenses to increase for the foreseeable future as we continue to invest in R&D activities related to developing product candidates, including investments in manufacturing, as

our programs advance into later stages of development and as we conduct additional clinical trials.

The process of conducting the necessary clinical research to obtain regulatory approval is costly and time-consuming, and the successful

development of product candidates is highly uncertain.

As a result, we are unable to determine the duration and completion costs of R&D projects or when and to what extent we will generate revenue from the commercialization and sale of any of

our product candidates.

We expect R&D expenses to increase for the foreseeable future as we continue to invest in R&D activities related to developing product candidates, including investments in manufacturing, as our

programs advance into later stages of development and as we conduct additional clinical trials.

The process of conducting the necessary clinical research to obtain regulatory approval is costly and time-consuming, and the successful development of

product candidates is highly uncertain.

As a result, we are unable to determine the duration and completion costs of R&D projects or when and to what extent we will generate revenue from the commercialization and sale of any of our product

Our future R&D expenses will depend on the clinical success of our product candidates, as well as ongoing assessments of the commercial potential of such product candidates.

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the development of our product candidates.

For example, if the FDA or another regulatory authority were to require us to conduct clinical trials or other testing beyond those that we currently contemplate for the completion of clinical

development of any of our product candidates that we may develop or if we experience significant delays in enrollment in any of our clinical trials, we could be required to expend significant additional financial resources and time on the

completion of clinical development of that product candidate.

For example, if the FDA or another regulatory authority were to require us to conduct clinical trials or other testing beyond those that we currently contemplate for the completion of clinical development

of any of our product candidates that we may develop or if we experience significant delays in enrollment in any of our clinical trials, we could be required to expend significant additional financial resources and time on the completion of

clinical development of that product candidate.

General and Administrative Expenses

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Interest Expense

Interest expense for the three months ended March 31, 2023 and 2022, related to our financing lease obligation for the Cranbury, NJ facility.

Interest expense for the three and six months ended June 30, 2023 and 2022 related to our financing lease obligation for the Cranbury, NJ facility.

Interest and Other Income

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Critical Accounting Policies and Significant Judgments and Estimates

There have been no material changes in our critical accounting policies and estimates in the preparation of our condensed consolidated financial statements during the three months ended March 31,

2023, compared to those disclosed in our 2022 Form 10-K.

There have been no material changes in our critical accounting policies and estimates in the preparation of our consolidated financial statements during the three months ended June 30, 2023, compared to

those disclosed in our 2022 Form 10-K.

Results of Operations

Comparison of the Three Months Ended March 31, 2023 and 2022

Three Months Ended March 31,

Comparison of the Three Months Ended June 30, 2023 and 2022

Three Months Ended June 30,

Operating expenses:

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Research and Development Expenses

R&D expenses increased $15.6 million to $46.4 million for the three months ended March 31, 2023 compared to the three months ended March 31, 2022.

The increase in R&D expenses was primarily

driven by increases in manufacturing and development costs of $2.8 million, compensation and benefits of $6.6 million due to increased R&D headcount, direct materials of $0.9 million, and laboratory supplies of $0.9 million.

R&D expenses increased $10.0 million to $51.4 million for the three months ended June 30, 2023 compared to the three months ended June 30, 2022.

The increase in R&D expenses was primarily driven

by increases in costs for compensation and benefits of $5.7 million due to increased R&D headcount, clinical trial costs of $2.7 million, non-cash stock compensation expense of $1.7 million.

General and Administrative Expenses

G&A expenses increased $4.1 million to $15.8 million for the three months ended March 31, 2023, compared to the three months ended March 31, 2022.

The increase in G&A expenses was primarily

driven by increases in commercial preparation related expenses of $1.1 million, compensation and benefits of $0.7 million due to increased G&A headcount and non-cash stock compensation expense of $1.1 million.

G&A expenses increased $4.5 million to $17.4 million for the three months ended June 30, 2023, compared to the three months ended June 30, 2022.

The increase in G&A expenses was primarily driven

by increases in commercial preparation related expenses of $1.3 million, compensation and benefits of $0.8 million due to increased G&A headcount and non-cash stock compensation expense of $1.1 million.

Other Income (Expense), Net

Other income increased $4.3 million to $3.9 million for the three months ended March 31, 2023, compared to the three months ended March 31, 2022.

The increase in other income was primarily driven by

an increase in interest and other income, net, of $1.3 million and an increase in accretion of discount and amortization of premium on investments, net, of $3.0 million.

The increase in interest and other income, net, of $1.3 million was due to

increased interest rates of $0.7 million and reduced fair value of warrant liability of $0.7 million.

Other income increased $3.2 million to $3.1 million for the three months ended June 30, 2023, compared to the three months ended June 30, 2022.

The increase in other income was primarily driven by an

increase in accretion of discount and amortization of premium on investments, net, of $3.1 million and an increase in interest and other income, net, of $0.2 million.

The increase in interest and other income, net, of $0.2 million was primarily due

to increased interest rates of $0.3 million and partially offset by an increased fair value of warrant liability of $0.2 million.

Comparison of the Six Months Ended June 30, 2023 and 2022

Six Months Ended June 30,

Operating expenses:

Research and development

General and administrative

Total operating expenses

Loss from operations

Interest expense

Interest and other income, net

Accretion of discount and amortization of premium on investments - net

Total other income (expense), net

Research and Development Expenses

R&D expenses increased $25.6 million to $97.8 million for the six months ended June 30, 2023 compared to the six months ended June 30, 2022.

The increase in R&D expenses was primarily driven by

increases in costs for compensation and benefits of $12.3 million due to increased R&D headcount, clinical trial costs of $3.4 million, non-cash stock compensation expense of $3.2 million, increases in manufacturing and development costs of

$1.6 million, direct materials of $1.4 million, and laboratory supplies of $1.4 million.

General and Administrative Expenses

G&A expenses increased $8.6 million to $33.2 million for the six months ended June 30, 2023, compared to the six months ended June 30, 2022.

The increase in G&A expenses was primarily driven by

increases in commercial preparation related expenses of $2.4 million, compensation and benefits of $1.6 million due to increased G&A headcount and non-cash stock compensation expense of $2.3 million.

Other Income (Expense), Net

Other income increased $7.5 million to $6.9 million for the six months ended June 30, 2023, compared to the six months ended June 30, 2022.

The increase in other income was primarily driven by an

increase in accretion of discount and amortization of premium on investments, net, of $6.1 million and interest and other income, net, of $1.5 million.

The increase in interest and other income, net, of $1.5 million was due to increased interest

rates of $1.1 million and reduced fair value of warrant liability of $0.5 million.

Liquidity and Capital Resources

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government regulations and the need to obtain additional financing.

Drug candidates currently under development will require significant additional R&D efforts, including extensive preclinical and clinical testing and regulatory approval,

prior to commercialization.

Drug candidates currently under development will require significant additional R&D efforts, including extensive preclinical and clinical testing and regulatory approval, prior

to commercialization.

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

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has incurred net losses and negative cash flows from its operations each year since inception.

Rocket incurred net losses of $58.3 million for the three months ended March 31, 2023, and $221.9 million for the year ended December 31, 2022.

experienced negative cash flows from operations and as of March 31, 2023 and December 31, 2022, we had an accumulated deficit of $772.1 million and $713.8 million, respectively.

As of March 31, 2023, we had $360.0 million of cash, cash

equivalents and investments.

Rocket incurred net losses of $124.0 million for the six months ended June 30, 2023, and $221.9 million for the year ended December 31, 2022.

experienced negative cash flows from operations and as of June 30, 2023 and December 31, 2022, we had an accumulated deficit of $837.8 million and $713.8 million, respectively.

As of June 30, 2023, we had $307.0 million of cash, cash equivalents

and investments.

We expect such resources will be sufficient to fund our operating expenses and capital expenditure requirements into the first half of 2025.

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In the longer term, our future viability is dependent on our ability to generate cash from operating activities or to raise additional capital to finance our operations.

If we raise additional funds

by issuing equity securities, our stockholders will experience dilution.

If we raise additional funds by

issuing equity securities, our stockholders will experience dilution.

Any future debt financing into which we enter may impose upon us additional covenants that restrict our operations, including limitations on our ability to incur liens or

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Our failure to raise capital as and when needed could have a negative impact on our financial condition and ability to pursue our business strategies.

Three Months Ended March 31,

Six Months Ended June 30,

Net cash used in operating activities

Net cash used in investing activities

Net cash (used in) provided by investing activities

Net cash provided by financing activities

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Operating Activities

During the three months ended March 31, 2023, operating activities used $57.6 million of cash and cash equivalents, primarily resulting from our net loss of $58.3 million offset by net non-cash

charges of $8.2 million, including non-cash stock-based compensation expense of $8.9 million, accretion of discount on investments of $2.3 million, and depreciation and amortization expense of $1.7 million.

Changes in our operating assets and

liabilities for the three months ended March 31, 2023 consisted of a decrease in accounts payable and accrued expenses of $7.8 million, a decrease in our prepaid expenses of $0.9 million, and a decrease in other liabilities of $0.7 million.

During the three months ended March 31, 2022, operating activities used $39.2 million of cash, primarily resulting from our net loss of $43.0 million offset by net non-cash

charges of $8.2 million, including non-cash stock-based compensation expense of $6.3 million, accretion of discount on investments of $0.6, and depreciation and amortization expense of $1.3 million.

During the six months ended June 30, 2023, operating activities used $108.4 million of cash and cash equivalents, primarily resulting from our net loss of $124.0 million offset by net non-cash charges

of $17.7 million, including non-cash stock-based compensation expense of $19.2 million, depreciation and amortization expense of $3.4 million, partially offset by accretion of discount on investments of $4.8 million.

Changes in our operating assets

and liabilities for the six months ended June 30, 2023 included a decrease in accounts payable and accrued expenses of $4.5 million, a decrease in our prepaid expenses of $2.7 million, and a decrease in other liabilities of $0.5 million.

During the six months ended June 30, 2022, operating activities used $78.3 million of cash, primarily resulting from our net loss of $97.4 million offset by net non-cash

charges of $17.7 million, including non-cash stock-based compensation expense of $13.6 million, amortization of premium on investments of $1.0, and depreciation and amortization expense of $2.8 million.

Changes in our operating assets and

liabilities for the three months ended March 31, 2022, consisted of a decrease in accounts payable and accrued expenses of $0.5 million and a decrease in our prepaid expenses of $3.9 million.

liabilities for the six months ended June 30, 2022, included an increase in accounts payable and accrued expenses of $2.5 million and an increase in our prepaid expenses of $1.1 million.

Investing Activities

During the three months ended March 31, 2023, net cash used by investing activities was $36.7 million, primarily resulting from proceeds of $62.3 million from the maturities of investments, offset by

During the six months ended June 30, 2023, net cash used in investing activities was $5.5 million, primarily resulting from proceeds of $170.6 million from the maturities of investments, offset by

purchases of investments of $168.9 million, and purchases of property and equipment of $7.1 million.

During the three months ended March 31, 2022, investing activities used $63.0 million of cash, primarily resulting from proceeds of $82.0 million from the maturities of investments, offset by

During the six months ended June 30, 2022, net cash provided by investing activities was $15.8 million, primarily resulting from proceeds of $163.7 million from the maturities of investments, offset by

purchases of investments of $143.0 million, and purchases of property and equipment of $4.9 million.

Financing Activities

During the three months ended March 31, 2023, financing activities provided $18.3 million of cash, primarily resulting from net proceeds of $17.2 million from the sale of shares through our

at-the-market facility.

During the three months ended March 31, 2022, net cash provided by financing activities was $0.1 million, consisting of the issuance of common stock, pursuant to exercises of stock options.

During the six months ended June 30, 2023, net cash provided by financing activities was $18.5 million, consisting primarily of proceeds of $17.2 million from the sale of shares through our

at-the-market facility and $1.3 million from the exercise of stock options.

During the six months ended June 30, 2022, net cash provided by financing activities was $17.3 million, consisting primarily of proceeds of $17.2 million from the issuance the common stock in connection

with the at-the-market offering program.

Contractual Obligations and Commitments

There were no material changes outside the ordinary course of our business to the contractual obligations specified in the table of contractual obligations included in “Management’s Discussion and

Analysis of Financial Condition and Results of Operations” in our 2022 Form 10-K.

Information regarding contractual obligations and commitments may be found in Note 12 of our unaudited consolidated financial statements in this Quarterly Report on

We do not have any off-balance sheet arrangements that are material or reasonably likely to become material to our financial condition or results of operations.

Information regarding contractual obligations and commitments may be found in Note 12 of our unaudited consolidated financial statements in this Quarterly Report on Form 10-Q.

We do not have any

off-balance sheet arrangements that are material or reasonably likely to become material to our financial condition or results of operations.

Recently Issued Accounting Pronouncements

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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 2. Management’s Discussion and Analysis