Rocket Pharmaceuticals Announces European Medicines Agency Acceptance of RP-L102 Marketing Authorization Application for the Treatment of Fanconi Anemia
Rocket Pharmaceuticals, Inc. (RCKT)
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Source: Business Wire
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.--(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 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.“The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to
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