CRANBURY, N.J. / Aug 20, 2025 / 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 that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease. The... Read More

Prioritizing development of RP-A501 (Danon disease), RP-A601 (PKP2-ACM), and RP-A701 (BAG3-DCM) to advance AAV cardiovascular gene therapy platform for sustained value creation FDA RMAT designation awarded to RP-A601 for PKP2-ACM; Engaging with FDA on pivotal trial design following encouraging initial Phase 1 data at ASGCT IND accepted and FDA Fast Track designation received for RP-A701 in BAG3-DCM program; Phase 1 trial... Read More

Restructuring pipeline to focus on AAV cardiovascular platform (Danon disease, PKP2-ACM, BAG3-DCM) and regulatory activities for KRESLADI™ (severe LAD-I), setting foundation for near- and long-term value creation Organizational restructuring expected to reduce headcount by approximately 30% and lower 12-month cash burn by nearly 25% Company expects current cash runway to fund operations into the second quarter of 2027... Read More

CRANBURY, N.J. / Jul 17, 2025 / 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 that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601, the Company’s investigational... Read More

RP-A701 is a first-in-class gene therapy for the treatment of BAG3-associated dilated cardiomyopathy BAG3-associated dilated cardiomyopathy is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality Rocket plans to conduct a Phase 1 trial in the U.S. and is working towards the first... Read More

Conference call to be held later today at 8:30 a.m. ET CRANBURY, N.J. / May 27, 2025 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing an integrated pipeline of genetic therapies for rare diseases, today announced an update related to RP-A501, its investigational gene therapy for Danon disease. A patient participating in the Phase 2 pivotal trial of... Read More

RP-A601 was generally well-tolerated at a dose of 8.0E13 GC/kg with no dose-limiting toxicities in all three patients with up to 12 months follow-up RP-A601 promoted increased protein expression and desmosomal localization of Plakophilin-2 (PKP2), Desmocollin-2, and Cadherin-2 in all three patients Improvement or stabilization observed in arrhythmia burden, heart function, and quality of life in all patients No further dose... Read More

CRANBURY, N.J. / May 09, 2025 / 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 that the company will be presenting data for the first time from the Phase 1 clinical trial of RP-A601 to treat patients with plakophilin-2 related arrhythmogenic... Read More

Phase 2 pivotal study of RP-A501 for Danon disease is ongoing; Program update expected mid-year 2025 Initial data from the Phase 1 study of RP-A601 for PKP2-ACM anticipated May 2025 IND submission for BAG3-DCM program expected mid-year 2025 Sarbani Chaudhuri appointed Chief Commercial & Medical Affairs Officer Cash, cash equivalents and investments of approximately $318.2M; expected operational runway into the fourth quarter... Read More

CRANBURY, N.J. / Apr 03, 2025 / 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 that management will participate in the 24th Annual Needham Virtual Healthcare Conference taking place on April 8, 2025. Gaurav Shah, M.D., Chief Executive Officer, will... Read More

Dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease; Program update expected in the first half of 2025 New England Journal of Medicine publication of Phase 1 study of RP-A501 to treat patients with Danon disease Long-term data from the Phase 1 trial of RP-A501 for Danon disease presented at AHA on safety and meaningful efficacy Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for... Read More

CRANBURY, N.J. / Jan 02, 2025 / 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 that the Company will participate in the 43rd Annual J.P. Morgan Healthcare Conference, in San Francisco, California. Gaurav Shah, M.D., Chief Executive Officer, is... Read More

CRANBURY, N.J. / Dec 11, 2024 / 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, 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... Read More

CRANBURY, N.J. / Dec 10, 2024 / 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, 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... Read More

CRANBURY, N.J. / Nov 26, 2024 / 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 that the Company will participate in the 7 th Annual Evercore HealthCONx Conference in Miami, Florida. Gaurav Shah, M.D., Chief Executive Officer, will take part in a... Read More

All six evaluable Phase 1 patients with Danon disease are alive and transplant-free up to age 25 years RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10% reduction in LV mass index at 12 months and sustained through most recent follow up (up to five years) First patient treated shows preliminary evidence of robust protein expression in heart on five-year biopsy... Read More

CRANBURY, N.J. / Nov 12, 2024 / 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 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... Read More

CRANBURY, N.J. / Nov 08, 2024 / 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 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... Read More

CRANBURY, N.J. / Sep 17, 2024 / 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 that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease. After the two-patient... Read More

CRANBURY, N.J. / Sep 10, 2024 / 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 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... Read More

CRANBURY, N.J. / Aug 27, 2024 / 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 that the Company will participate in the Morgan Stanley 22 nd Annual Global Healthcare Conference and the 2024 Cantor Fitzgerald Global Healthcare Conference in New York.... Read More

Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat PKP2 arrhythmogenic cardiomyopathy Working toward FDA-approval of KRESLADI for severe LAD-I; Commercial infrastructure and capabilities in place for launch Presented long-term KRESLADI ™ follow-up data from the global Phase 1/2 study for severe LAD-I, results from the global... Read More

CRANBURY, N.J. / Jun 28, 2024 / 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 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... Read More

CRANBURY, N.J. / May 29, 2024 / 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 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... Read More

Long-term KRESLADI TM 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 profile in 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... Read More

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,... Read More

Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined with hematologic stabilization extending out to 42 months after treatment with RP-L102 CRANBURY, N.J. / Apr 02, 2024 / Business Wire / Rocket Pharmaceuticals, Inc . (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare... Read More

Aaron Ondrey joins as Chief Financial Officer bringing seasoned leadership experience in financial management, strategic planning, commercial finance, and capital allocation Additional key appointments within existing corporate leadership team expand capabilities and expertise spanning early discovery through commercialization CRANBURY, N.J. / Mar 26, 2024 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a... Read More

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... Read More

New Prescription Drug User Fee Act (PDUFA) date of June 30, 2024 CRANBURY, N.J. / Feb 13, 2024 / 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 the U.S. Food and Drug Administration (FDA) has extended the Priority Review period for the Biologics... Read More

CRANBURY, N.J. / Jan 02, 2024 / 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 that Gaurav Shah, M.D., Chief Executive Officer, is scheduled to present at the 42 nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 3:45 p.m. PT.... Read More

CRANBURY, N.J. / Dec 12, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

Agreement reached with FDA on Phase 2 pivotal study design of RP-L301 for PKD to support accelerated approval; Initiating single-arm, 10-patient pivotal study with primary endpoint of ≥1.5 point Hgb improvement at 12 months Initiated Phase 2 pivotal trial of RP-A501 for Danon Disease following FDA alignment Initiated Phase 1 study of RP-A601 for PKP2-ACM FDA accepted BLA with Priority Review for KRESLADI TM (marnetegragene... Read More

CRANBURY, N.J. / Oct 19, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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 30 th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Brussels, Belgium, taking place October 24-27. Updated data will... Read More

Prescription Drug User Fee Act (PDUFA) target action date is March 31, 2024 RP-L201 has received FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, Fast Track and Orphan Drug designations; Rocket eligible for Priority Review Voucher, if RP-L201 is approved CRANBURY, N.J. / Oct 02, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an... Read More

CRANBURY, N.J. / Sep 26, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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 7 th Annual Genetic Medicines Conference in New York... Read More

CRANBURY, N.J. / Sep 13, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

Final alignment reached on a 12 patient, single-arm, open label study with a biomarker based co-primary endpoint assessed at 12 months to support accelerated approval Co-primary endpoint consisting of LAMP2 protein expression and Left Ventricular (LV) Mass change from baseline CRANBURY, N.J. / Sep 12, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an... Read More

CRANBURY, N.J. / Sep 12, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

CRANBURY, N.J. / Sep 06, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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 21 st Annual Global Healthcare Conference and Cantor Fitzgerald Global Healthcare Conference 2023.... Read More

Initiating enrollment of two-patient pediatric safety run-in for pivotal single-arm Phase 2 study of RP-A501 (Danon Disease); finalizing primary composite endpoint with FDA to support accelerated approval RP-L201 (LAD-I) BLA submitted to FDA; represents Rocket’s first product filing RP-A601 (PKP2-ACM) advancing towards first patient treatment in Phase 1 study following FDA clearance of IND; received FDA Fast Track and Orphan... Read More

CRANBURY, N.J. / Jun 08, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

CRANBURY, N.J. / May 31, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

CRANBURY, N.J. / May 23, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

Robust and sustained efficacy in both adult PKD patients up to 30 months post-RP-L301; first pediatric patient results suggest efficacy similar to adult cohort with initial greater than five-point increase in hemoglobin Sustained genetic correction observed in eight of 12 evaluable Fanconi Anemia patients, and phenotypic correction and concomitant hematologic stabilization observed in seven of 12 patients 12-42 months after... Read More

First clinical gene therapy program for PKP2-ACM, a devastating inherited heart disease affecting approximately 50,000 people in the U.S. and EU Robust preclinical proof of concept studies showed RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model Initiating Phase 1 trial start-up activities and rapidly working towards first patient treatment CRANBURY, N.J. / May 09, 2023 / Business Wire /... Read More

Continued to advance leading pipeline of AAV cardiology and LV hematology assets; all 2023 milestones remain on track Updates on all four clinical programs and PKP2-ACM preclinical program to be presented at the 26 th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) Mark White, MB.ChB, named Chief Medical Officer following more than 25 years at AstraZeneca; Jonathan Schwartz, M.D., appointed Chief Gene... Read More

CRANBURY, N.J. / May 03, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. ( NASDAQ: RCKT), 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 Bank of America Securities 2023 Healthcare Conference in Las... Read More

CRANBURY, N.J. / Apr 11, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

Jonathan Schwartz, M.D., appointed Chief Gene Therapy Officer to enhance the Company’s focus on strategic application of gene therapy technologies to current and future therapeutic areas Mark White, MB.ChB, named Chief Medical Officer; Dr. White brings proven track record in late-stage development and commercialization with more than 25 years of experience at AstraZeneca CRANBURY, N.J. / Apr 04, 2023 / Business Wire / Rocket... Read More

CRANBURY, N.J. / Feb 07, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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... Read More

RP-A601 for Arrhythmogenic Cardiomyopathy due to PKP2 pathogenic variants (PKP2-ACM) represents meaningful commercial opportunity with estimated prevalence of 50,000 adults and children in the U.S. and EU; IND filing anticipated in Q2 2023 In support of Phase 2 pivotal study, latest positive Phase 1 data in Danon Disease recently shared with FDA demonstrate improvements in all biomarker and clinical endpoints across... Read More

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), 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 41 st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at 2:15 p.m. PT. A live audio webcast of the... Read More

Based on ongoing FDA discussion, Rocket to pursue a biomarker-based composite endpoint for a single arm, open-label pivotal trial with a natural history comparator Initiation of initial component of global study anticipated in 1H’23 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with... Read More

Sustained genetic and phenotypic correction and concomitant hematologic stabilization observed in at least six of 10 evaluable FA patients between 12 and 36 months after RP-L102; a seventh patient potentially demonstrates engraftment and stabilization after 36 months Robust and sustained efficacy in both adult PKD patients at 24 months post-RP-L301 demonstrated by normalized hemoglobin and improved hemolysis parameters... Read More