Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease

This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.

Start Date05 Sep 2023

Sponsor / Collaborator

Rocket Pharmaceuticals, Inc.

NCT03882437 / Active, not recruitingPhase 1

A Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With DD

This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene (investigational product (IP), RP-A501) in male patients with Danon Disease (DD).

Start Date17 Apr 2019

Sponsor / Collaborator

Rocket Pharmaceuticals, Inc.

100 Clinical Results associated with RP-A501

100 Translational Medicine associated with RP-A501

100 Patents (Medical) associated with RP-A501

News (Medical) associated with RP-A501

06 May 2024

·www.biospace.com

Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

Advanced RP-L102 for Fanconi Anemia towards regulatory reviews; EMA accepted MAA for review and BLA submission anticipated in the first half of 2024 Continued preparations to launch LV portfolio beginning with KRESLADI™ (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024 Appointed Aaron Ondrey as Chief Financial Officer, bringing seasoned leadership experience in commercial-stage financial management, strategic planning, and capital allocation Cash, cash equivalents and investments of approximately $330.3M; expected operational runway into 2026 CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending March 31, 2024. “Rocket has had a strong start to 2024. This quarter has been marked by meaningful progress across all our clinical programs, most notably with the EMA’s review of RP-L102 for Fanconi Anemia, commercial preparations for the potential approval of KRESLADI for severe LAD-I, and ongoing enrollment in the Phase 2, pivotal study of RP-A501 for Danon Disease and the Phase 1 study of RP-A601 for PKP2-ACM,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “As we build on the advancements made this quarter, we remain focused on execution for patients with rare and devastating diseases with limited treatment options.” Recent Pipeline and Operational Updates Advanced Fanconi Anemia (FA) program through key regulatory milestones. In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial. The Company remains on track to submit the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2024. KRESLADI™ on track for June 30, 2024, PDUFA date. Rocket continues to ramp up enabling activities to support the launch of its lentiviral (LV) vector portfolio beginning with KRESLADI™ for severe Leukocyte Adhesion Deficiency-I (LAD-I). Qualified Treatment Center initiation, disease education, payer engagement, and field team build-out are all underway. Bolstered finance, investor relations, and corporate communications expertise of company leadership to support evolution towards commercial stage. Rocket appointed Aaron Ondrey as Chief Financial Officer (CFO). Mr. Ondrey brings over 20 years of experience leading commercial-stage financial management, strategic planning, and capital allocation. Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals. In addition, Meg Dodge was appointed Vice President to lead Investor Relations & Corporate Communications. Ms. Dodge is experienced with engaging stakeholders across investors, media, and other communities in the biotech and financial sectors. Prior to joining Rocket, Ms. Dodge was Head of Investor Relations and Corporate Communications at Krystal Biotech. Celebrated annual Rare Disease Day with multi-faceted awareness campaign. On February 29, 2024, Rocket hosted its annual Rare Disease Day recognition program highlighting the theme, “Leap into Action for Rare.” Several hundred attendees gathered at the Liberty Science Center and virtually to hear inspirational stories from the community. Rocket also continued its Light Up for Rare initiative in collaboration with global partners to light up buildings and landmarks in Rare Disease Day colors, including the Empire State Building. Rocket remains highly committed to supporting the rare disease community through patient-focused events, education and advancing science to bring potential treatments to patients with unmet needs. Milestones in 2024 are on track across its pipeline of gene therapies for rare and devastating diseases. Rocket continues to advance three disclosed programs from its adeno-associated virus (AAV) cardiovascular portfolio, including: Phase 2 pivotal study of RP-A501 for Danon Disease, Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM) In its late-stage LV portfolio, Rocket is working towards initiation of the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD). Data from Rocket’s LV hematology portfolio to be presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting. Updated data across Rocket’s LV hematology programs will be highlighted as oral presentations at the ASGCT 27th Annual Meeting taking place May 7-11, 2024, in Baltimore, MD. Incremental updates include longer-term data demonstrating the safety and efficacy of Rocket’s Phase 1/2 pivotal studies of KRESLADI™ for severe LAD-I and RP-L102 for FA, in addition to the Phase 1 study of RP-L301 for PKD. Details for oral presentations are as follows: Title: Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301 Session: Clinical Trials Spotlight Symposium Presenter: Julián Sevilla, M.D., Ph.D., Clinical Investigator, Hematología y Hemoterapia, Hematología y Oncología Pediátricas, Hospital Infantil Universitario Niño Jesús Presentation date and time: Wednesday, May 8, 2024, 8:00 a.m. – 8:15 a.m. ET Location: Ballroom 1 Presentation number: 4 Title: Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning Session: Cell Therapy and Cell-Based Gene Therapy Trials Presenter: Agnieszka Czechowicz, M.D., Ph.D., Center for Definitive and Curative Medicine, Department of Pediatrics, Division of Hematology/ Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Lucile Packard Children’s Hospital Stanford Presentation date and time: Friday, May 10, 2024, 2:45 p.m. – 3:00 p.m. ET Location: Ballroom 1 Presentation number: 245 Title: Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Favorable Safety Pro /> Session: Cell Therapy and Cell-Based Gene Therapy Trials Presenter: Donald B. Kohn, M.D., Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy Program, University of California, Los Angeles Presentation date and time: Friday, May 10, 2024, 3:00 p.m. – 3:15 p.m. ET Location: Ballroom 1 Presentation number: 246 Upcoming Investor Conference Bank of America Global Healthcare Conference 2024: May 16, 2024 First Quarter Financial Results Cash position. Cash, cash equivalents and investments as of March 31, 2024, were $330.3 million. R&D expenses. Research and development expenses were $45.2 million for the three months ended March 31, 2024, compared to $46.4 million for the three months ended March 31, 2023. The decrease in R&D expenses was primarily driven by decreases in manufacturing and development costs and direct materials of $5.8 million. Decreases were partially offset by increases in costs for compensation and benefits expense of $1.4 million due to increased R&D headcount, professional fees of $1.1 million, laboratory supplies of $0.9 million, non-cash stock compensation expense of $0.8 million, and clinical trial costs of $0.6 million. G&A expenses. General and administrative expenses were $22.1 million for the three months ended March 31, 2024, compared to $15.8 million for the three months ended March 31, 2023. The increase in G&A expenses was primarily driven by increased commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $3.3 million, legal expenses of $1.5 million, and non-cash stock compensation expense of $0.5 million. Net loss. Net loss was $62.1 million or $0.66 per share (basic and diluted) for the three months ended March 31, 2024, compared to $58.3 million or $0.73 (basic and diluted) for the three months ended March 31, 2023. Shares outstanding. 90,646,590 shares of common stock were outstanding as of March 31, 2024. Financial Guidance Cash position. As of March 31, 2024, Rocket had cash, cash equivalents and investments of $330.3 million. Rocket expects such resources will be sufficient to fund its operations into 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “will give,” “estimate,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its DD program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2023, filed February 27, 2024 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Three Months Ended March 31, 2024 2023 Operating expenses: Research and development $ 45,227 $ 46,371 General and administrative 22,148 15,823 Total operating expenses 67,375 62,194 Loss from operations (67,375 ) (62,194 ) Interest expense (471 ) (468 ) Interest and other income, net 3,029 1,908 Accretion of discount on investments, net 2,763 2,419 Net loss $ (62,054 ) $ (58,335 ) Net loss per share - basic and diluted $ (0.66 ) $ (0.73 ) Weighted-average common shares outstanding - basic and diluted 93,549,884 79,453,519 March 31, 2024 December 31, 2023 Cash, cash equivalents, and investments $ 330,313 $ 407,495 Total assets 499,442 566,341 Total liabilities 57,940 73,767 Total stockholders' equity 441,502 492,574

Phase 1Executive ChangeFinancial StatementPhase 2

11 Mar 2024

·www.biospace.com

Regulatory Pathway Widening for Cardiac Gene Therapy Hopefuls

Pictured: An FDA blue van drives towards cardiac-shaped mountains/ Nicole Bean for BioSpace After five gene therapies hit the market last year, FDA officials have predicted that 2024 will be a “breakout year” for approvals of this type of treatment. With more than 17 gene therapies now available for patients, cardiovascular indications—which have recently seen increased activity—are navigating largely uncharted regulatory waters. While recent approvals can help guide regulators in learning the new modalities of gene therapies, the cardiovascular landscape is still emerging. The arena is essentially split between companies focusing on highly prevalent diseases and those working on rare cardiovascular disorders, experts told BioSpace, and each comes with its own regulatory challenges. Leveraging Biomarker Flexibility A handful of biotech companies are targeting rare diseases that affect the heart. Because many of these diseases lack effective therapies, new treatments can take advantage of the FDA’s accelerated approval pathway. In general, gene therapies for orphan indications are 3.5 times more likely than average drugs to be approved once in a Phase I trial, Tim Hunt, CEO of the Alliance for Regenerative Medicine (ARM), said during the annual Cell & Gene State of the Industry Briefing in January. Lexeo Therapeutics is developing a gene therapy, LX2006, for Friedreich’s ataxia cardiomyopathy, for which the FDA has granted Rare Pediatric Disease designation and Orphan Drug designation. Historically, cardiovascular (CV) treatments have relied upon large population and long-term studies with endpoints of mortality, morbidity and hospitalization, Lexeo CEO R. Nolan Townsend told BioSpace. But Townsend sees a shift occurring, which could open the door to a precision medicine approach for gene therapies in the cardiac space. “Cardiac is beginning to look a lot more like some of the rare neurologic diseases . . . in terms of the flexibility that the FDA is giving companies to have these medicines reach patients,” he said. A lot of this flexibility stems from a better understanding of the biomarkers associated with rare CV diseases, Townsend said, adding that using biomarkers as a surrogate endpoint allows companies to evaluate efficacy at earlier stages and often in smaller patient populations—a crucial component for targeting rare diseases. In its Friedreich’s ataxia program, Lexeo is using both invasive and non-invasive biomarkers pretreatment and post-treatment to evaluate the one-time gene therapy’s efficacy. Townsend said he hopes the biomarker data will lead to an accelerated approval. Rocket Pharmaceuticals is also utilizing biomarkers in the testing of its gene therapy for Danon disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle that often results in heart failure. After entering the clinic in 2018, the company began pivotal trials of RP-A501 last year. Sami Corwin, a William Blair analyst who covers Rocket, credits the faster-than-usual development timeline to the smaller patient population required by the FDA, in addition to the use of biomarkers. “The FDA has seemed to be a little more lenient in terms of pivotal trial design,” Corwin told BioSpace, adding that the required duration of follow-up is shorter for trials of gene therapies for rare diseases compared to those assessing therapies for a highly prevalent CV disease. The shorter follow-up times, smaller patient populations and use of biomarkers to prove early efficacy are helping to de-risk gene therapy programs for investors, allowing smaller biotech companies to compete in the space, Corwin said. Ultimately, there are a number of cardiovascular diseases that may not be large enough to attract big pharma that small-to-midsized companies can now tackle due to this increased flexibility, Townsend said. “I think we will see a broadening in the number of cardiovascular precision medicines over the coming years based on this evolving regulatory trend.” Large Indications Face Greater Scrutiny In the other camp, some gene therapy players are targeting CV indications with large populations, such as levels of bad cholesterol. “Regulators are clearly looking at rare diseases with really no other therapeutic alternative as a completely different regulatory landscape to more prevalent disorders,” Miles Minter, an analyst with William Blair covering Verve Therapeutics, told BioSpace. Indications for which treatments are already approved are treated differently, he said. Verve is developing a one-time gene therapy to lower cholesterol, but to be FDA approval-worthy, it will need to work in the most severe patients, Minter said. Companies like Verve need to show their therapy is effective in these patients before moving to earlier lines of treatment. However, “Gene editing could completely transform how we treat cardiovascular disease, shifting the paradigm from daily pills or intermittent injections over decades to potentially a once-and-done [solution],” Verve CEO Sekar Kathiresan told BioSpace in an email. Kathiresan said understanding FDA expectations is critical for Verve’s programs. VERVE-101 turns off the PCSK9 gene in the liver to lower LDL cholesterol. The program is partnered with Eli Lilly, potentially pointing to the need for big pharma collaboration to meet trial design requirements for a large population indication. Delivery Modality Matters Most CV-indicated gene therapies are delivered via one of two delivery modalities, Corwin said. The genetic materials for those targeting more prevalent indications like Verve’s VERVE-101 are being delivered via the liver through a lipid nanoparticle (LNP). But for indications that need to change proteins within the heart itself, adeno-associated virus (AAV) vectors are the delivery method of choice. Corwin said the regulatory pathway is likely more complicated with AAV. The FDA is tuned in to the potential toxicity risks of AAV gene therapies, having hosted an advisory committee meeting on the subject in 2021. The manufacturing process for AAV is also complex, expensive and time-intensive. Corwin and Minter both agreed that the agency is likely more comfortable with LNP-delivered therapies, thanks to the widespread use of mRNA-based COVID-19 vaccines with LNP vectors. “There are different nuances and points that the FDA will scrutinize” when it comes to CV-indicated gene therapies, Corwin said. As of press time, the FDA had not responded to BioSpace’s request for comment on regulatory requirements for CV-indicated gene therapies. For CV indications common and rare, Townsend extolled gene therapy’s potential. “At the moment, I don’t want to say it’s the only game in town, but it is probably the best game in town for delivering a nucleic acid to the heart.” Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

Gene TherapyOrphan DrugVaccine

26 Feb 2024

·www.biospace.com

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Advancing leading pipeline of six disclosed programs across AAV cardiovascular and LV hematology portfolios; all milestones remain on track for 2024 Expanding commercial capabilities to support launch of LV portfolio beginning with KRESLADI™ (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024 Cash, cash equivalents and investments of approximately $407.5M; expected operational runway into 2026 CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023. “I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “We are well poised to make further strides in 2024, notably expanding our commercial and operational capabilities to support the anticipated launch of KRESLADI™ (marnetegragene autotemcel) for severe LAD-I, representing Rocket’s first step in making our gene therapies available to patients who need them most. At the same time, we are laser focused on regulatory filings in Fanconi Anemia and continuing to advance our clinical programs in Danon Disease, PKP2-ACM and PKD in the year ahead. As we close the quarter, Rocket continues to solidify our industry leadership in gene therapy across multiple therapeutic areas and platforms as we seek to meet the needs of patients living with rare and devastating diseases.” Recent Pipeline and Operational Updates Milestones remain on track for 2024 across pipeline of adeno-associated virus (AAV) cardiovascular and lentiviral (LV) vector hematology portfolios. The Marketing Authorisation Application (MAA) and Biologics License Application (BLA) for Fanconi Anemia remain on track for filing with the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA), respectively, in the first half of 2024. Rocket is also progressing its Phase 2 pivotal study of RP-A501 for Danon Disease, Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD), Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM). KRESLADI™ for severe Leukocyte Adhesion Deficiency-I (LAD-I) on track for PDUFA date of June 30, 2024. Based on the positive top-line efficacy and safety data from the global Phase 1/2 study and high unmet need in patients with severe LAD-I, the FDA accepted the BLA and granted Priority Review for KRESLADI™. On February 13, 2024, Rocket announced the FDA extended the Priority Review period by three months, to June 30, 2024, to allow additional time to review clarifying Chemistry, Manufacturing, and Controls (CMC) information submitted by Rocket in response to FDA information requests. The FDA further confirmed that an advisory committee meeting is not needed. In support of its first product launch, Rocket continues to expand its commercial and operational infrastructure, including Qualified Treatment Center initiation, channel strategy, disease education, and payer engagement. Published data from preclinical study of RP-A601 in PKP2-ACM. “AAV-Mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans” was published in Circulation: Genomic and Precision Medicine. Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM. Bolstered commercial and operational expertise of Board of Directors with appointment of R. Keith Woods. Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain. He recently served as Chief Operating Officer of argenx, where he led its global commercial organization, including marketing, market access, medical affairs, program management, and supply chain, during the company’s successful transition to commercial stage. Mr. Woods launched Vyvgart, a treatment for a rare autoimmune condition that causes muscle weakness, generating more than $1 billion in sales. Upcoming Investor Conferences Cowen’s 44th Annual Health Care Conference: March 5, 2024 Leerink Partners Global Biopharma Conference: March 12, 2024 Needham 23rd Annual Virtual Healthcare Conference: April 9, 2024 Fourth Quarter and Full Year 2023 Financial Results Cash position. Cash, cash equivalents and investments as of December 31, 2023, were $407.5 million. R&D expenses. Research and development expenses were $41.7 million and $186.3 million for the three and twelve months ended December 31, 2023, respectively, compared to $50.0 million and $165.6 million for the three and twelve months ended December 31, 2022, respectively. The increase in R&D expenses for the twelve months ended December 31, 2023, was primarily driven by increased compensation and benefits expense of $16.9 million due to increased R&D headcount, increased clinical trial costs of $14.5 million, and increased non-cash stock compensation expense of $5.0 million, offset by a decrease in manufacturing and development costs of $17.0 million. G&A expenses. General and administrative expenses were $21.5 million and $73.3 million for the three and twelve months ended December 31, 2023, respectively, compared to $19.0 million and $58.8 million for the three and twelve months ended December 31, 2022, respectively. The increase in G&A expenses for the twelve months ended December 31, 2023, was primarily driven by increases in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $8.4 million and increased non-cash stock compensation expense of $3.4 million. Net loss. Net loss was $59.7 million and $245.6 million or $0.64 and $2.92 per share (basic and diluted) for the three and twelve months ended December 31, 2023, compared to $66.7 million and $221.9 million or $0.92 and $3.26 per share (basic and diluted) for the three and twelve months ended December 31, 2022. Shares outstanding. 90,282,267 shares of common stock were outstanding as of December 31, 2023. Financial Guidance Cash position. As of December 31, 2023, Rocket had cash, cash equivalents and investments of $407.5 million. Rocket expects such resources will be sufficient to fund its operations into 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket’s lentiviral (LV) vector-based gene therapies target hematologic diseases and consist of late-stage programs for Fanconi Anemia, a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Our adeno-associated virus (AAV)-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. For more information about Rocket, please visit and follow us on LinkedIn, YouTube and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its DD program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2022, filed February 28, 2023 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Three Months Ended December 31, Twelve Months Ended December 31, 2023 2022 2023 2022 Operating expenses: Research and development $ 41,744 $ 50,037 $ 186,342 $ 165,570 General and administrative 21,535 19,044 73,317 58,773 Total operating expenses 63,279 69,081 259,659 224,343 Loss from operations (63,279 ) (69,081 ) (259,659 ) (224,343 ) Research and development incentives - 500 - 500 Interest expense (470 ) (467 ) (1,875 ) (1,862 ) Interest and other income, net 814 1,245 5,288 3,889 Accretion of discount and amortization of premium on investments, net 3,275 1,081 10,651 (47 ) Net loss $ (59,660 ) $ (66,722 ) $ (245,595 ) $ (221,863 ) Net loss per share - basic and diluted $ (0.64 ) $ (0.92 ) $ (2.92 ) $ (3.26 ) Weighted-average common shares outstanding - basic and diluted 93,336,541 72,889,548 84,009,004 68,148,925 December 31, December 31, 2023 2022 Cash, cash equivalents, and investments $ 407,495 $ 399,670 Total assets 566,341 551,807 Total liabilities 73,767 62,121 Total stockholders' equity 492,574 489,686

Phase 2Phase 1Gene TherapyFinancial Statement

100 Deals associated with RP-A501

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Glycogen Storage Disease Type IIb	Phase 2	US	Rocket Pharmaceuticals, Inc.	05 Sep 2023
Diverticular Diseases	Phase 1	US	Rocket Pharmaceuticals, Inc.	17 Apr 2019

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Phase 1 RP-A501 (ASGCT2023)	Phase 1	Glycogen Storage Disease Type IIb \| Cardiomyopathies LAMP2 gene mutations \| LAMP2 protein deficiency	7	RP-A501 low dose	ziqwkhjfwd(hwaupizuml) = All RP-A501- or IM-related adverse events (AEs) were manageable and reversible. A single RP-A501-related SAE of grade 4 thrombotic microangiopathy and renal failure requiring transient dialysis (with full recovery from both) was reported in 1 adult pt in the high dose cohort. No RP-A501 or steroid-related SAEs have been observed to date in the pediatric pts. cczwobhmbo (sjatcdskue ) View more	Positive	01 May 2023

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