Rocket Pharmaceuticals

194 articles about Rocket Pharmaceuticals

• Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia

  4/2/2024

  Rocket Pharmaceuticals, Inc. announced that the European Medicines Agency accepted the Marketing Authorization Application for RP-L102, its lentiviral vector-based investigational gene therapy for Fanconi Anemia, complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterized by bone marrow failure, cancer predisposition, and congenital malformations.

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  6 Recently Approved Cell & Gene Therapies—And Why They Matter

  4/1/2024

  With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.

• Rocket Pharmaceuticals Announces Appointment of Aaron Ondrey as Chief Financial Officer and Additional Updates to Corporate Leadership Team

  3/26/2024

  Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) 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

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

  2/26/2024

  Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

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  FDA Pushes Back PDUFA Data for Rocket’s Gene Therapy for Rare Blood Disorder

  2/14/2024

  Citing the need for more time to review additional Chemistry, Manufacturing and Controls information, the FDA has extended its target action date for Rocket Pharmaceuticals’ investigational gene therapy by three months.

• Rocket Pharmaceuticals Announces Update on FDA Review Timeline of KRESLADI™ (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

  2/13/2024

  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 License Application (BLA) for KRESLADI™ (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I).

• Rocket Pharmaceuticals to Present at 42nd Annual J.P. Morgan Healthcare Conference

  1/2/2024

  Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) 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 Appoints R. Keith Woods to Board of Directors

  12/12/2023

  Rocket Pharmaceuticals, Inc. today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors.

• Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

  11/6/2023

  Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) 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.

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  Study Highlights Standout Clinical Success of Certain Cell and Gene Therapies

  11/6/2023

  A recent report by Tufts Medical Center reveals impressive approval rates for durable cell and gene therapies, but experts urge caution over persistent safety and accessibility concerns.

• Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)

  10/19/2023

  Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral and AAV Gene Therapies at the 30th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT).

• Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)

  10/2/2023

  Rocket Pharmaceuticals Announces FDA Acceptance of Biologics License Application with Priority Review for RP-L201 (marnetegragene autotemcel) for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I).

• Rocket Pharmaceuticals to Participate in the Chardan 7th Annual Genetic Medicines Conference

  9/26/2023

  Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) 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 Announces Pricing of Public Offering of Common Stock - September 13, 2023

  9/13/2023

  Rocket Pharmaceuticals, Inc. announced the pricing of an underwritten public offering of 7,812,500 shares of its common stock at a public offering price of $16.00 per share and 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.

• Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease

  9/12/2023

  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 alignment has been reached with the Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of RP-A501 for Danon Disease.

• Rocket Pharmaceuticals Announces Proposed Public Offering of Common Stock - September 12, 2023

  9/12/2023

  Rocket Pharmaceuticals, Inc. announced 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.

• Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences

  9/6/2023

  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 21st Annual Global Healthcare Conference and Cantor Fitzgerald Global Healthcare Conference 2023.

• Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress

  8/10/2023

  Rocket Pharmaceuticals, Inc. 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 Receives FDA Fast Track and Orphan Drug Designations for RP-A601 Gene Therapy for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

  6/8/2023

  Rocket Pharmaceuticals, Inc. 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 Receives European Medicines Agency (EMA) Priority Medicines (PRIME) Designation for RP-A501 Gene Therapy for Danon Disease

  5/31/2023

  Rocket Pharmaceuticals, Inc. 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.