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Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review.

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 (LAD-I).

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 Pharmaceuticals, Inc. 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.

Rocket Pharmaceuticals, Inc. announced longer-term data updates from its lentiviral vector hematology portfolio presented at the 27th Annual Meeting of the American Society of Gene and Cell Therapy.

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

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, 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.

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.

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