FANCA

Long-term KRESLADITM follow-up data demonstrate survival of 100% in the absence of allogeneic hematopoietic stem cell transplantation (HSCT) from 18 to 45 months with a well-tolerated safety pro all nine patients with severe LAD-I Previously disclosed results from the global RP-L102 Fanconi Anemia Phase 1/2 trial demonstrate genetic and phenotypic correction combined with hematologic stabilization extending to 42 months with polyclonal integration patterns Sustained and clinically meaningful hemoglobin improvement and well-tolerated safety pro PKD patients up to 36 months after RP-L301 treatment 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 announced longer-term data updates from its lentiviral (LV) vector hematology portfolio presented at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Data updates demonstrate the continued safety and efficacy of the Phase 1/2 pivotal studies of KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) and RP-L102 for Fanconi Anemia (FA), in addition to the Phase 1 study of RP-L301 for Pyruvate Kinase Deficiency (PKD). “The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals. “Ahead of the upcoming PDUFA date, KRESLADITM continues to demonstrate 100% HSCT-free survival and significant reductions in infection-related hospitalizations following engraftment in patients with severe LAD-I. In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization. Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients. We are very pleased with the safety pro across our LV hematology portfolio, with no drug-related serious adverse events observed to date.” Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Well-Tolerated Safety Pro > The oral presentation includes positive, updated data (cut-off July 24, 2023) from the global Phase 1/2 pivotal studies demonstrating sustained efficacy and safety of KRESLADITM from 18 to 45 months of follow-up for all nine patients with severe LAD-I. Observed 100% survival in the absence of allogeneic HSCT at least 18 months post-infusion in all nine patients; all patients enrolled at less than 12 months of age have surpassed 24 months without HSCT. All primary and secondary endpoints were met, including sustained genetic and phenotypic correction. When compared with pre-treatment history, data showed substantial decreases in the incidences of significant infections requiring hospitalization or intravenous antimicrobials, combined with evidence of resolution of LAD-I-related skin and periodontal lesions and restoration of wound repair capabilities. KRESLADITM was well-tolerated in all patients with no drug-related serious adverse events reported to date. Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and are consistent with the safety profiles of those agents and procedures. No cases of graft failure or autologous graft-versus-host-disease (GvHD) were reported. Based on the positive efficacy and safety data from the global pivotal studies of KRESLADITM for severe LAD-I, the Biologics License Application (BLA) was accepted for review by the U.S. Food and Drug Administration (FDA) who has set the New Prescription Drug User Fee Act (PDUFA) target date of June 30, 2024. Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning The oral presentation includes positive, updated data (cut-off September 11, 2023) from the global Phase 1/2 pivotal studies of RP-L102, Rocket’s ex vivo LV gene therapy candidate for FA. Consistent with results observed from the clinical program, RP-L102 is a potentially curative therapy to prevent FA-related bone marrow failure (BMF), which can be administered without a suitable allogeneic donor or transplant-related toxicities. RP-L102 demonstrates sustained and progressively increasing genetic correction in eight of 12 patients with greater than 12 months of follow-up. Observed genetic correction is associated with phenotypic correction and hematologic stability. RP-L102 remains well-tolerated with no significant safety signals. For the first time, data demonstrate that RP-L102 confers polyclonal insertion patterns indicative of long-term hematopoietic stem cell repopulation of the bone marrow and peripheral blood and clonal diversity in the absence of conditioning. Based on the positive efficacy and safety data from the global pivotal studies of RP-L102 for FA, the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for review. Rocket expects to submit the BLA to the FDA in the first half of 2024. Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301 The oral presentation includes positive, updated data (cut-off February 5, 2024) from the Phase 1 study from two adult patients with PKD treated with RP-L301 followed up to 36 months and two pediatric patients followed up to 12 months. Sustained and clinically meaningful hemoglobin improvement observed in all patients including hemoglobin normalization in three of four patients. No patients have required red blood cell transfusion following neutrophil engraftment. Improvements in hemoglobin supported by improved markers of hemolysis and quality of life have been observed. RP-L301 remains well-tolerated, with no drug-related serious adverse events. Insertion site analyses in the peripheral blood and bone marrow for both adult patients through 36 months post-RP-L301 demonstrated highly polyclonal patterns with no clonal dominance or insertional mutagenesis. Testing is ongoing for pediatric patients who were more recently treated. Based on the positive safety and efficacy data from the global Phase 1 study of RP-L301 for PKD, Rocket is working towards initiation of the Phase 2 pivotal study. About KRESLADITM (marnetegragene autotemcel) KRESLADITM is an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I) that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designations in both regions for the program. KRESLADITM was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. The LV vector was developed in a collaboration between University College London and CIEMAT. About Leukocyte Adhesion Deficiency-I Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, survival beyond childhood is rare. Currently the only potential curative treatment is an allogeneic hematopoietic stem cell transplant (HSCT), which may not be available in time for these children and itself has substantial morbidity and mortality. There is a high unmet medical need for patients with severe LAD-I. Rocket’s LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California. About RP-L102 RP-L102 is an investigational gene therapy for Fanconi Anemia (FA) that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to contain a functional copy of the FANCA gene. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. RP-L102 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. About Fanconi Anemia Fanconi Anemia (FA) is a rare genetic disorder characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations. In the absence of allogeneic hematopoietic stem cell transplant (HSCT), the primary cause of death among patients with FA is BMF, which typically occurs during the first decade of life. Allogeneic HSCT, when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Both chemotherapy conditioning and graft-versus-host disease, a known complication of allogeneic HSCT, are associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene lead to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a “gold standard” test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of an FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as “natural gene therapy” provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells. There is a high unmet medical need for patients with FA. About RP-L301 RP-L301 is an investigational gene therapy that contains autologous hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vector to contain a functional copy of the PKLR gene, which is responsible for energy production in red blood cells (RBCs). RBCs carry oxygen to the rest of the body. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations in the U.S., EMA PRIME designation in the EU, and Orphan Drug designation in both regions for the program. RP-L301 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz (IIS-FJD). About Pyruvate Kinase Deficiency Pyruvate Kinase Deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red blood cell destruction and potentially life-threatening anemia with a significant impact to quality of life. PKD has an estimated prevalence of 4,000 to 8,000 patients in the U.S. and Europe. Children are the most commonly and severely affected subgroup of patients. Patients with PKD have a high unmet medical need, as currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload. Mitapivat, an oral enzyme activator, is approved for use in adult patients, however its efficacy is limited in more severely-afflicted patients, most notably in those who are splenectomized, transfusion-dependent, or whose disease results from deleterious mutations. 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, Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies and Rocket’s ability to transition to a commercial stage pharmaceutical company. 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, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, 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. View source version on businesswire.com: Contacts Media & Investors Meg Dodge Media Kevin Giordano media@rocketpharma.com Investors Brooks Rahmer investors@rocketpharma.com Source: Rocket Pharmaceuticals, Inc. View this news release online at:

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

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。美国FDA批准强生与传奇生物联合开发的嵌合抗原受体（CAR）-T细胞疗法Carvykti用于二线治疗复发性或难治性多发性骨髓瘤（RRMM）成年患者。百时美施贵宝和2seventy bio 联合开发的CAR-T疗法Abecma也获得美国FDA的批准，治疗更早期的多发性骨髓瘤患者。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF———✦研发进展✦———◇美国FDA批准强生（Johnson & Johnson）与传奇生物（Legend Biotech）联合开发的嵌合抗原受体（CAR）T疗法Carvykti（ciltacabtagene autoleucel）用于治疗复发性或难治性多发性骨髓瘤（RRMM）成年患者，这些患者之前曾至少接受过包括蛋白酶体抑制剂（PI）和免疫调节剂（IMiD）的一线治疗，并对来那度胺（lenalidomide）耐药。根据新闻稿，Carvykti是首个获批用于多发性骨髓瘤患者二线或以上治疗的B细胞成熟抗原（BCMA）靶向疗法。此次批准主要基于CARTITUDE-4研究数据，这是一项国际、随机、开放标签的3期研究，用于评估Carvykti对比标准治疗方案（SOC）在既往接受过1-3线治疗的来那度胺耐药的RRMM成人患者中的疗效和安全性。研究中的SOC包括泊马度胺、硼替佐米和地塞米松（PVd）或达雷妥尤单抗、泊马度胺和地塞米松（DPd）。根据FDA所公布的ODAC会议相关文件显示，根据独立审查委员会（IRC）评估，CARTITUDE-4试验中使用Carvykti治疗患者的无进展生存期（PFS）与标准治疗组相比具有统计显著性改善（HR=0.41，95% CI：0.30-0.56，p<0.0001）。◇百时美施贵宝（Bristol Myers Squibb）和2seventy bio宣布，美国FDA基于KarMMa-3临床试验的结果，批准靶向BCMA的CAR-T细胞疗法Abecma（idecabtagene vicleucel）用于治疗复发或难治性多发性骨髓瘤成年患者，这些患者在接受包括IMiD、PI和抗CD38单克隆抗体的两种或更多先前治疗后疾病出现进展。此次批准扩大了Abecma的适应症范围，让它可以治疗更早期的多发性骨髓瘤患者。这一批准是基于3期临床试验KarMMa-3的积极结果。中位随访时间为15.9个月时，与标准方案相比，Abecma将主要终点中位PFS提高了三倍以上，达到13.3个月（95% CI：11.8-16.1），活性对照组为4.4个月（95% CI：3.4-5.9），两者具统计学差异（HR：0.49；95% CI：0.38-0.64；p<0.0001）。这表明使用Abecma治疗将疾病进展或死亡的风险降低了51%。◇Kyverna Therapeutics公司宣布，其靶向CD19的候选CAR-T疗法KYV-101在两名进展性多发性硬化（MS）患者中的研究结果在Med杂志上发表。新闻稿指出，这是KYV-101首次被用于治疗多发性硬化患者。试验结果显示，KYV-101表现出可接受的安全性特征，未发现早期神经毒性的临床征兆。在患者脑脊液中观察到CAR-T细胞的存在和扩增，并且在一名患者中观察到鞘内抗体水平的下降，这意味着CAR-T细胞可以靶向中枢神经系统中的CD19阳性B细胞。研究人员表示，这一结果支持在更为大型的临床试验中评估KYV-101。KYV-101是一款自体、全人源CD19靶向CAR-T细胞疗法，用于治疗B细胞驱动的自身免疫疾病。它目前正在1/2期临床试验中接受评估，用于治疗狼疮性肾炎患者。治疗多发性硬化和重症肌无力的IND申请也已经获得FDA的许可。◇LyGenesis公司宣布，首位终末期肝病（ESLD）患者已在该公司的2a期临床试验中接受潜在“first-in-class”再生细胞疗法的移植。这一疗法将健康供体的肝细胞移植到ESLD患者的淋巴结中，目标是在淋巴结中生长出一个或多个迷你肝脏。这些迷你肝脏能够弥补受损肝脏的功能，挽救终末期肝病患者的生命。新闻稿指出，这是首次在临床试验中利用患者自身的淋巴结作为“培养箱“来生长异位器官，代表着再生医学领域的重要里程碑。这项2a期临床试验计划招募12名ESLD患者，每位接受细胞移植的患者将接受随访1年，评估移植的同种异体肝细胞的安全性、耐受性和疗效。新闻稿指出，这一疗法可以仅用一个捐献肝脏生产的细胞，就可以治疗数十名ESLD患者，有望缓解目前移植器官短缺的问题。◇Ring Therapeutics公司宣布，该公司在预印本网站bioRxiv上发表了基于其Anellogy技术平台开发的基因疗法的临床前研究。新闻稿指出，这是首个基于人类共生病毒——指环病毒（anellovirus）的基因递送载体系统。它的独特性在于载体不会激发人体免疫反应，所以具备重复给药的潜力。这项研究描述了如何将转基因载荷装进指环病毒的衣壳，以及基于指环病毒载体的基因疗法的体外和体内实验结果。体外实验验证了基于指环病毒的载体可以转染视网膜色素上皮（RPE）细胞。在动物实验中，基于指环病毒的载体与AAV9在注射到视网膜下后达到的表达水平相似。图片来源：123RF◇Rocket Pharmaceuticals公司宣布，欧洲药品管理局（EMA）已经接受该公司为基因疗法RP-L102递交的上市申请，用于治疗特定范可尼贫血（FA）。范可尼贫血是一种罕见遗传疾病，主要特征为骨髓衰竭、癌症风险高和先天性畸形。60~70%的范可尼贫血患者携带FANCA基因突变，它编码在DNA修复中至关重要的蛋白。FANCA基因的突变导致染色体断裂和细胞对氧化和环境应激因子的敏感性。RP-L102是一款在研基因疗法，使用慢病毒载体将FANCA基因的功能性拷贝递送到患者自身的造血干细胞中。它已经获得美国FDA授予的再生医学先进疗法认定和快速通道资格，以及EMA授予的PRIME认定。◇Caribou Biosciences宣布，为同种异体CD19靶向CAR-T疗法CB-010递交的IND申请已经获得美国FDA的许可，用于治疗狼疮性肾炎（LN）和肾外狼疮（ERL）。预计CB-010治疗LN和ERL患者1期临床试验GALLOP将于2024年底前启动。CB-010是Caribou公司的同种异体CAR-T细胞疗法平台产出的主要临床阶段候选疗法。先前的报告显示，CB-010在1期临床试验ANTLER的剂量递增部分已显示出鼓舞人心的初步安全性和有效性。该试验针对的是复发或难治性B细胞非霍奇金淋巴瘤（r/r B-NHL）患者。与许多自体和异体CAR-T细胞疗法不同，CB-010的制造不依赖于慢病毒或其他逆转录病毒载体。Caribou公司的chRDNA基因编辑技术允许在T细胞DNA的预定位置精确插入表达嵌合抗原受体的转基因。———✦融资、合作、M&A✦———◇Obsidian Therapeutics宣布完成1.605亿美元C轮融资。获得资金将用于推进Obsidian主打肿瘤浸润淋巴细胞（TIL）疗法OBX-115的开发，该项目正在进行针对黑色素瘤和非小细胞肺癌患者的1/2期临床试验。OBX-115是一种新型工程化的TIL疗法，该TIL具有表达于细胞膜上受调节的白介素-15（IL-15）蛋白，因此患者在接受OBX-115治疗时不需要同时接受高剂量的白介素-2（IL-2）治疗。接受传统TIL治疗的患者需同时接受具潜在毒性的IL-2蛋白以活化体内的TIL。临床前数据显示，与未经改造的TIL疗法相比，OBX-115具有增强的持久性与效力，动物模型的肿瘤控制得到改善，因此有潜力改善不同类型实体瘤患者的临床结果。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] IECURE ANNOUNCES FDA CLEARANCE OF INVESTIGATIONAL NEW DRUG APPLICATION FOR ECUR-506 TO INITIATE OTC-HOPE TRIAL FOR TREATMENT OF NEONATAL ONSET ORNITHINE TRANSCARBAMYLASE DEFICIENCY IN THE U.S. Retrieved April 4, 2024, from https://iecure.com/news/iecure-announces-fda-clearance-of-investigational-new-drug-application-for-ecur-506-to-initiate-otc-hope-trial-for-treatment-of-neonatal-onset-ornithine-transcarbamylase-deficiency-in-the-u-s/[2] In major step toward the clinic, Ring’s human viral vector delivers gene therapy to eyes of mice. Retrieved April 4, 2024, from https://www.fiercebiotech.com/research/major-step-towards-clinic-rings-new-human-viral-vector-delivers-gene-therapy-eyes-mice[3] Myrtelle's rAAV-Olig001-ASPA Gene Therapy Candidate for Canavan Disease Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from the U.S. Food and Drug Administration. Retrieved April 4, 2024, from https://www.prnewswire.com/news-releases/myrtelles-raav-olig001-aspa-gene-therapy-candidate-for-canavan-disease-receives-regenerative-medicine-advanced-therapy-rmat-designation-from-the-us-food-and-drug-administration-302104076.html[4] Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia. Retrieved April 4, 2024, from https://www.businesswire.com/news/home/20240402101364/en[5] Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease. Retrieved April 4, 2024, from https://www.globenewswire.com/news-release/2024/04/01/2855565/0/en/Ocugen-Announces-Positive-Data-and-Safety-Monitoring-Board-Review-and-Initiation-of-Enrollment-in-Medium-Dose-for-OCU410ST-a-Modifier-Gene-Therapy-in-GARDian-Study-for-Stargardt-Di.html[6] Ring Therapeutics Publishes a Novel Gene Delivery Platform Based on the Commensal Human Anellovirus. Retrieved April 4, 2024, from https://www.globenewswire.com/news-release/2024/04/01/2855145/0/en/Ring-Therapeutics-Publishes-a-Novel-Gene-Delivery-Platform-Based-on-the-Commensal-Human-Anellovirus.html[6] Obsidian Therapeutics Announces Oversubscribed $160.5 Million Series C Financing to Drive OBX-115 Clinical Development. Retrieved April 4, 2024, from https://www.businesswire.com/news/home/20240403825922/en[7] LyGenesis Announces First Patient Dosed in its Phase 2a Clinical Trial of a First-in-Class Regenerative Cell Therapy for Patients with End-Stage Liver Disease. Retrieved April 4, 2024, from https://www.prnewswire.com/news-releases/lygenesis-announces-first-patient-dosed-in-its-phase-2a-clinical-trial-of-a-first-in-class-regenerative-cell-therapy-for-patients-with-end-stage-liver-disease-302105734.html[8] First-in-Disease Use of Kyverna Therapeutics' KYV-101 in Patients With Progressive Multiple Sclerosis Published in Med. Retrieved April 4, 2024, from https://www.prnewswire.com/news-releases/first-in-disease-use-of-kyverna-therapeutics-kyv-101-in-patients-with-progressive-multiple-sclerosis-published-in-med-302103748.html免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新