Articles from Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, announced today the pricing of an upsized underwritten public offering of 13,200,000 shares of its common stock at a public offering price of $12.50 per share. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $165 million. All shares in the offering are to be sold by Rocket. In addition, Rocket has granted the underwriters a 30-day option to purchase up to an additional 1,980,000 shares of its common stock offered in the public offering. The offering is expected to close on or about December 12, 2024, subject to customary closing conditions.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, announced today that it intends to offer and sell $150 million of shares of its common stock in an underwritten public offering. In addition, Rocket intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of shares of its common stock offered in the public offering. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the 7th Annual Evercore HealthCONx Conference in Miami, Florida. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, December 3, at 1:45 p.m. ET and host investor meetings throughout the day.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, presented long-term safety and efficacy results from the Phase 1 RP-A501 study which showed that RP-A501 was generally well tolerated and all evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of left ventricular (LV) mass index by ≥10% at 12 months (sustained up to 54 months) after treatment. These data were presented today at a Late-Breaking Scientific session at the American Heart Association (AHA) Scientific Sessions 2024, published in The New England Journal of Medicine (NEJM) and discussed on a company webinar today at 12:00 p.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the company will be presenting new data on the results of the RP-A501 Phase 1 trial to treat patients with Danon disease at the American Heart Association’s 2024 Late-Breaking Science sessions being held from November 16-18, 2024 in Chicago, Illinois.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the UBS Global Healthcare Conference in Rancho Palos Verdes, CA. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, November 12, at 5:00 p.m. PT.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), 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 third quarter ending September 30, 2024.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of Mikael Dolsten, M.D., Ph.D., as an independent director to its Board of Directors. Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the Morgan Stanley 22nd Annual Global Healthcare Conference and the 2024 Cantor Fitzgerald Global Healthcare Conference in New York. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat and management will host investor meetings at each conference. Participation details are as follows:

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), 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 June 30, 2024.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet needs, today announced a regulatory update for KRESLADI™ (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I). The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to Rocket’s Biologics License Application for KRESLADI™ wherein the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Commission (EC), based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted orphan medicinal product designation for RP-A601, the Company’s adeno-associated virus (AAV.rh74)-based gene therapy candidate for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), 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).

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), 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 Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102, its lentiviral (LV) vector-based investigational gene therapy for Fanconi Anemia (FA), complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure (BMF), cancer predisposition, and congenital malformations.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of Aaron Ondrey, as Chief Financial Officer, and the further evolution of its corporate leadership team through key appointments with existing team members. These leadership announcements build on and expand Rocket’s preparedness for its planned transition to commercial stage and continued development of its pipeline.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), 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.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the U.S. Food and Drug Administration (FDA) has extended the Priority Review period for the Biologics License Application (BLA) for KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I). The FDA extended the 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 has further confirmed that an advisory committee meeting is not needed.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to present at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 3:45 p.m. PT.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors. Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending September 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced presentations at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27. Updated data will be presented from the Phase 1 clinical trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Previously disclosed data will be presented from the Phase 2 pivotal trial of RP-L102 for Fanconi Anemia, Phase 1 trial of RP-A501 for Danon Disease and preclinical studies supporting the Phase 1 trial of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM). Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, Rocket Pharma, will also give an Invited Talk about the importance of collaboration with and commitment to patients in gene therapy development.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) and granted Priority Review for RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. The PDUFA date set by the FDA is March 31, 2024.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to participate in a fireside chat at the Chardan 7th Annual Genetic Medicines Conference in New York City on Tuesday, October 3, at 1:00 p.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of (i) 7,812,500 shares of its common stock at a public offering price of $16.00 per share and (ii) to certain investors, pre-funded warrants to purchase 3,126,955 shares of common stock at a price of $15.99 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.01 per share exercise price for each such pre-funded warrant. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be approximately $175 million. All shares and pre-funded warrants in the offering are to be sold by Rocket. In addition, Rocket has granted the underwriters a 30-day option to purchase up to an additional 1,640,918 shares of its common stock. The offering is expected to close on or about September 15, 2023, subject to customary closing conditions.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today that it intends to offer and sell shares of its common stock, and to certain investors, pre-funded warrants to purchase shares of its common stock, in an underwritten public offering. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $150 million. The purchase price of each pre-funded warrant will equal the price per share at which shares of common stock are being sold to the public in this offering, minus $0.01, which will be the per share exercise price of each pre-funded warrant. All shares and pre-funded warrants in the offering are to be sold by Rocket. In addition, Rocket intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of shares of its common stock offered in the public offering. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that alignment has been reached with the Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of RP-A501 for Danon Disease. Danon Disease is a uniformly fatal inherited cardiomyopathy that leads to mortality in the majority of male patients at age ~20 and females at age ~40, and for which there are no approved curative or disease-modifying therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Company management will participate in the Morgan Stanley 21st Annual Global Healthcare Conference and Cantor Fitzgerald Global Healthcare Conference 2023. Participation details are as follows:

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending June 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to RP-A601, the Company’s gene therapy candidate for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM).

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease, a devastating and fatal inherited cardiomyopathy for which there are no curative therapies. PRIME designation was granted based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A501 in patients with Danon Disease and the potential of RP-A501 to meet the high unmet medical need in this population.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the Company’s investigational lentiviral-based gene therapy for Pyruvate Kinase Deficiency (PKD), a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based on robust safety and efficacy data from the ongoing Phase 1 RP-L301 clinical trial and its potential to cure a life-threatening disease for which no curative therapies currently exist. The designation will provide the benefits of added FDA guidance and expedited review through the program’s development.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced positive data from the Pyruvate Kinase Deficiency (PKD), Fanconi Anemia, Severe Leukocyte Adhesion Deficiency-I (LAD-I) and Danon Disease clinical programs and PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) preclinical program presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM) a devastating inherited heart disease that can lead to life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death. The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative. Even with treatment, life-threatening arrhythmias and progression of disease may still occur. PKP2-ACM affects approximately 50,000 people in the U.S. and Europe.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending March 31, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Company management will participate in the 22nd Annual Needham Virtual Healthcare Conference and Chardan’s 7th Annual Genetic Medicines and Cell Therapy Manufacturing Summit. Participation details are as follows:

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced expansion of its leadership team to support its growing and industry-leading pipeline of AAV and LV gene therapy assets and evolution towards commercial stage. Jonathan Schwartz, M.D., Rocket’s founding Chief Medical Officer, has been appointed to the newly created position of Chief Gene Therapy Officer to expand the Company’s focus on the strategic application of gene therapy technologies to current and future therapeutic areas. In addition, Mark White, MB.ChB, joins Rocket as Chief Medical Officer following more than 25 years at AstraZeneca where he gained deep expertise in late-stage drug development and commercialization across small and large molecules, vaccines and therapeutic areas including rare diseases.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to participate in the Genetic Medicines Corporate Panel at the 43rd Annual Cowen Health Care Conference taking place in Boston, MA on Monday, March 6, 2023 at 10:30 a.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and 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, 2022.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to participate in a virtual fireside chat at the SVB Securities Global Biopharma Conference on Thursday, February 16, at 2:20 p.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A501 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program’s development.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces the addition of RP-A601 to Rocket’s cardiac gene therapy portfolio as well as anticipated highlights for the year ahead across the Company’s world-class pipeline of lentiviral and AAV gene therapy programs targeting rare hematologic and cardiovascular diseases. These announcements and anticipated highlights will be presented at the 41st Annual J.P. Morgan Healthcare Conference today at 2:15 p.m. PT by Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to present at the 41st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at 2:15 p.m. PT.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces updates from the Company's end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) regarding RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease. During the meeting, Rocket 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.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces positive clinical data from its lentiviral (LV)-based gene therapy programs at the 64th American Society of Hematology (ASH) Annual Meeting, taking place in New Orleans, Louisiana, from December 10-13.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that it has completed the previously announced acquisition of Renovacor, Inc. NYSE: RCORNYSERCOR)

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the 5th Annual Evercore ISI HealthCONx Conference on Tuesday, November 29, 2022, at 3:30 p.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today reports financial results for the quarter ending September 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces upcoming data presentations at the 75th American Heart Association (AHA) Annual Meeting, taking place in Chicago, Illinois, and virtually, from Nov. 5-7, 2022, and the 64th American Society of Hematology (ASH) Annual Meeting, taking place in New Orleans, Louisiana, and virtually, from Dec. 10-13, 2022. At AHA, the Company will share previously disclosed data from the Phase 1 trial of RP-A501, its adeno-associated virus (AAV)-based gene therapy for Danon Disease, in an oral presentation. During ASH, the Company will present poster presentations sharing data from its lentiviral vector (LV)-based gene therapy programs. Updated safety and efficacy data will be presented from the Phase 2 pivotal trial of RP-L102 for Fanconi Anemia and Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Previously disclosed data will be presented from the Phase 2 pivotal trial of RP-L201 for Leukocyte Adhesion Deficiency-I.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rocket’s lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announced that Raj Prabhakar, M.B.A., Chief Business Officer, will deliver an in-person company presentation at the 2022 Cell & Gene Meeting on the Mesa on Tuesday, October 11, at 2:30 p.m. PT in Carlsbad, CA.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of 6,800,000 shares of its common stock at a public offering price of $14.75 per share. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $100.3 million. All shares in the offering are to be sold by Rocket. In addition, Rocket has granted the underwriters a 30-day option to purchase up to an additional 1,020,000 shares of its common stock. The offering is expected to close on or about October 6, 2022, subject to customary closing conditions.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today that it has commenced an underwritten public offering of $100 million of shares of its common stock. Rocket also intends to grant the underwriters a 30-day option to purchase up to an additional $15 million of shares of its common stock. All of the shares in the proposed offering are to be sold by Rocket.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, today announces positive clinical updates from its Phase 1 Danon Disease Trial for RP-A501 through an oral poster session at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022. This includes updated safety and efficacy data from patients in the pediatric and adult cohorts which demonstrate that RP-A501 was generally well tolerated and conferred clinical benefit.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Chardan 6th Annual Genetic Medicines Conference on Tuesday, Oct. 4, at 2:00 p.m. ET, at the Westin Hotel in New York City. Management will also be hosting one-on-one meetings with investors.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that the Company will present updated clinical data on its Phase 1 Danon Disease Trial for RP-A501 during an oral poster session at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022 to be held Sept. 30 – Oct. 3, 2022. On the first day of the meeting at 6:00 a.m., the oral poster presentation will be made available on the congress online portal and the Company will issue a press release. An investor event will subsequently be webcast at 8:00 a.m. Rocket recently announced positive initial safety data from two pediatric patients in its Phase 1 Danon Disease trial at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Morgan Stanley 20th Annual Global Healthcare Conference on Monday, Sept. 13, at 3:30 p.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today reports financial results for the quarter ending June 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces the appointment of Mayo Pujols as Chief Technical Officer (CTO) and Executive Vice President. Mr. Pujols brings nearly three decades of experience, from leadership roles across technical operations, quality operations, validation, process development and Good Manufacturing Practice (cGMP) manufacturing. He most recently served as Chief Executive Officer of Andelyn Biosciences, a leading gene therapy contract development and manufacturing organization (CDMO) and prior to Andelyn was the Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals. As Rocket’s first CTO, Mr. Pujols will lead the technical operations function and chemistry, manufacturing and controls (CMC) for all lentiviral programs. Additionally, he will lead the Company’s state-of-the-art adeno-associated virus (AAV) manufacturing facility that has recently achieved cGMP readiness for a planned Phase 2 pivotal study in Danon Disease.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the William Blair Biotech Focus Conference 2022 on July 12-13, 2022, at the St. Regis Hotel in New York City. Management will also be hosting one-on-one meetings with investors.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces positive top-line safety and efficacy data from its Phase 2 pivotal trial for severe Leukocyte Adhesion Deficiency-I (LAD-I) at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces positive clinical updates from its Phase 2 pivotal trial for Fanconi Anemia (FA) and Phase 1 trials for Danon Disease and Pyruvate Kinase Deficiency (PKD) at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that Gaurav Shah, M.D., Chief Executive Officer, will deliver a company presentation at the UBS Global Healthcare Conference on Tuesday, May 24 at 7:45 a.m. ET, in New York.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today reports financial results for the quarter ending March 31, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, will deliver an in-person company presentation at the Bank of America Securities Healthcare Conference on Wednesday, May 11 at 2:20 p.m. ET at the Encore Hotel in Las Vegas.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that Kinnari Patel, Pharm.D., MBA, President and Chief Operating Officer, will deliver a virtual company presentation at the Guggenheim 3rd Annual Genomic Medicines and Rare Disease Conference on Friday, April 1 at 8:00 a.m. ET. Gaurav Shah, M.D., Chief Executive Officer, will present at the 21st Annual Needham Virtual Healthcare Conference on Monday, April 11 at 10:15 a.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Fady Malik, M.D., Ph.D., to its Board of Directors. Dr. Malik brings nearly 25 years of experience as an internationally recognized cardiovascular physician-scientist and highly successful biopharmaceutical executive. He will serve as an independent non-executive director.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual 11th Annual SVB Leerink Global Healthcare Conference on Wednesday, Feb. 16 at 10 a.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that Gaurav Shah, M.D., Chief Executive Officer, will deliver a company presentation virtually at the 40th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 10 at 1:30 p.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces positive clinical updates from its ongoing Phase 2 registrational trials for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) and its ongoing Phase 1 trial for Pyruvate Kinase Deficiency (PKD) at the 63rd American Society of Hematology (ASH) Annual Meeting.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President. Mr. Wilson brings nearly 20 years of legal, compliance and executive experience and accomplishment within the life sciences industry. Mr. Wilson will be responsible for the Legal and Compliance functions and serve as a key member of the leadership team.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host an in-person Investor and Analyst Event in Atlanta that will simultaneously be webcast on Tuesday, Dec. 14, 2021, at 7:30 a.m. ET to discuss updates from three of the Company’s lentiviral vector (LVV)-based gene therapy programs: RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I), RP-L102 for Fanconi Anemia (FA) and RP-L301 for Pyruvate Kinase Deficiency (PKD). Clinical data for all three trials will be presented at the 63rd American Society of Hematology (ASH) Annual Meeting being held Dec. 11-14, 2021.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the Evercore ISI 4th Annual HEALTHCONx Virtual Conference on Wednesday, Dec. 1, 2021 at 10:55 a.m. ET.

Rocket Pharmaceuticals, Inc. (NASDAQRCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces updated data from the Phase 1 clinical trial evaluating a single intravenous infusion of RP-A501, the Company’s investigational gene therapy, for the treatment of Danon Disease. This update, which includes interim safety and efficacy data from patients in the low-dose (6.7e13 vg/kg; n=3) and high-dose (1.1e14 vg/kg; n=2) adult and adolescent cohorts, demonstrates RP-A501 was generally well tolerated at the low-dose and conferred sustained clinical benefit.