Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association's 2024 Late-Breaking Scientific Sessions Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025 Rolling BLA submission initiated for RP-L102 for Fanconi Anemia Progress on FDA-approval of KRESLADI for severe LAD-I; Approval anticipated in 2025 Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors Cash, cash equivalents and investments of approximately $235.7M; expected operational runway into 2026

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 global pharmaceutical management experience. "As an industry-leading pharmaceutical executive, I am very pleased to welcome Mikael to our Board of Directors," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "His research and development experience will help advance our leading scientific capabilities and

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 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 Aaron Ondrey, as Chief Financial Officer, and the further evolution of its corporate leadership team through key appointments with existin

  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 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year

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 experience spanning commercialization, global operations, business strategy and supply chain. "I'm thrilled to welcome Keith to our Board of Directors, who brings deep industry expertise and invaluable experience to the Rocket Board as we build upon our commercial and operational infrastructure to support our anticipa

— Mayo Pujols brings nearly three decades of technical operations and manufacturing experience, of which the past five years have been exclusively focused on cell and gene therapy, from leading biopharma companies including Andelyn Biosciences, Novartis and Celgene— — Mr. Pujols will lead technical operations and the Company's state-of-the-art, recently cGMP ready, Clinical and potential Commercial AAV manufacturing facility in Cranbury, N.J. — Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), 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 appointmen

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), 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. "Fady is a tremendous addition to our Board of Directors, especially at this exciting time as we seek agency alignment on a Phase 2, potentially registration-enabling clinical study for Danon Disease, and continue to bui

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Jessie Yeung as Vice President of Investor Relations and Corporate Finance. Ms. Yeung brings more than 15 years of investor relations, corporate finance and capital market experience across industries including the biopharmaceutical and financial sectors. She will be responsible for leading the investor relations function and capital markets strategy. "I'm thrilled to welcome Jessie to Rocket as we continue to advance our world-class pipeline of gene therapy clinical programs and move towa

— Positive Phase 1 data from Danon Disease gene therapy trial demonstrated sustained benefit across clinical, functional and biomarker endpoints in all four adult patients with long-term follow up; pediatric cohort data expected Q3 2022 — — Reported engraftment in all nine severe LAD-I patients treated in pivotal Phase 2 trial and CD18 expression ranging from 26% to 87% of normal; top-line data expected Q2 2022— — Demonstrated evidence of engraftment in six Fanconi Anemia patients with at least 12-months of follow-up with MMC resistance between 16% and 63% at a minimum of one timepoint in pivotal Phase 2 trial; top-line data expected Q3 2022 — — Cash position of $388.7M; runway expected

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), 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. "The Rocket family is thrilled to welcome Martin to our talented executive leadership team as we advance our world-class pipeline of gene therapies and

4 - ROCKET PHARMACEUTICALS, INC. (0001281895) (Issuer)

4 - ROCKET PHARMACEUTICALS, INC. (0001281895) (Issuer)

Conference call to be held later today at 8:30 a.m. ET 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 RP-A501 experienced an unexpected Serious Adverse Event (SAE). The SAE involved clinical complications related to a capillary leak syndrome. Rocket is conducting a comprehensive root cause analysis and remains in active dialogue with the U.S. Food and Drug Administration (FDA) and other key stakeholders, with the c

— Reported positive safety data at ASGCT from pediatric cohort of Phase 1 Danon Disease study that demonstrated RP-A501 was well-tolerated; efficacy update across pediatric as well as adult cohorts on track for late Q3 2022— — Announced top-line data at ASGCT from pivotal Phase 2 trial for severe LAD-I that showed RP-L201 was well-tolerated with 100% overall survival at one year; regulatory filings anticipated in first half of 2023 — — Primary endpoint met for Fanconi Anemia pivotal Phase 2 trial, FDA dialogue initiated for BLA planning; continued data readouts for Fanconi Anemia and Pyruvate Kinase Deficiency programs expected in Q4 2022 — — Achieved Current Good Manufacturing Practice

— Continued to advance clinical gene therapy programs for the treatment of Danon Disease, Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) — — Updates on all four programs to be presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) this month; trials on track for 2022 data readouts — — Appointed internationally recognized cardiovascular physician-scientist and biopharmaceutical executive Fady Malik, M.D., Ph.D., to Board of Directors – — Named experienced commercial leader Carlos Martin to Chief Commercial Officer and proven biotech executive Jessie Yeung to Vice President of Investor Relations and C

—Results demonstrate sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up— — NYHA class improvement (from II to I) in all three patients (two low-dose, one high-dose) with closely monitored immunosuppressive regimen— —Decreased cardiac wall thickness with improved or stabilized ejection fraction on echocardiogram in all three patients with closely monitored immunosuppressive regimen— — Sustained improvement or stabilization in BNP and 6-minute walk test across four patients— —Sustained cardiac LAMP2B expression greater than 50% by immunohistochemistry and improved cardiac tissue architecture in all three patients with clos

—Company to provide comprehensive update on the ongoing Phase 1 clinical trial of RP-A501, including data to be presented at the American Heart Association (AHA) Scientific Sessions 2021— Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host a conference call and live webcast on Monday, Nov. 15, 2021, at 8:30 a.m. ET. Rocket will present updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease, which will include data being presented in an ePoster at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually Nov. 13-1

— Treatment initiated in pediatric patient cohort in Danon Disease trial of RP-A501— — Treatment completed in nine of nine patients in LAD-1 Phase 1/2 trial; favorable safety profile and initial clinical benefit reported in all seven patients with at least 3 months of follow up — — Clinical updates anticipated in Danon Disease at AHA 2021 Scientific Sessions and in FA, LAD-I and PKD at 63rd ASH Annual Meeting — — Ending Balance Sheet with $421.5 Million in Cash; Cash Runway Expected into 2H'23 — Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial r

— Working with FDA on Changes to Clinical Trial Protocol in Danon Disease Trial; Rocket Anticipates Trial Will Resume in 3Q — — Increasing and Durable Benefit Observed in Low Dose Cohort (6.7e13 dose) in Danon Disease; Removing Higher Dose Cohort (1.1e14 dose) From Future Dosing Plans; Full Clinical Update Expected in 4Q – — Positive Results from FA, LAD-I, PKD Trials Presented at 24th ASGCT Annual Meeting Show Preliminary Evidence of Activity and Favorable Tolerability — — Clinical Updates in FA, LAD-I, PKD and IMO Also Expected in 4Q — — Ending Balance Sheet with $426.8 Million in Cash; Cash Runway Expected into 2H'23 — — Conference Call to Be Held at 4:30 p.m. ET Today — Rocket Pha

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host a conference call and live audio webcast on Monday, Aug. 9, 2021, at 4:30 p.m. ET to report second quarter 2021 financial results and recent business highlights. Investors may access the conference call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550 from outside the United States. Please refer to conference ID number 50210581. A live webcast of the call can be accessed under "Events" in the Investors section of the Company's website at https://ir.rocketpharma.com/. The webcast

—RP-A501 Phase 1 Trial for the Treatment of Danon Disease Placed on Clinical Hold To Address FDA Request For Additional Risk Mitigation Methods in Protocol; No New Safety Events Observed; Anticipate Delay in Enrollment by One Quarter — —Updated Low Dose Results in RP-A501 Treatment for Danon Disease Demonstrate Stabilization or Improvements in Key Clinical Outcomes Including 6MWT and NYHA Class; Full Data to be presented in 4Q — — Reported Hemoglobin Increases of Nearly 2x from Baseline Sustained Up to 6-Months Post RP-L301 Treatment in PKD — — Announced Sustained Efficacy, Durability and Safety Up to 18-Months Post RP-L201 Treatment in LAD-I — — Advanced Fifth Gene Therapy Program in

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that it will host a webcast on Monday, December 7 at 6:00PM EST to discuss presentations at the 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs. Following the discussion, Rocket management and key opinion leaders will be available for a brief Q&A session. To access the li