Rocket Pharmaceuticals, Inc. 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. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial. RP-L102 demonstrated sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization.

Although allogeneic transplants can cure the hematologic component of FA, they confer significant side effects and substantially increase the risk of solid organ malignancies, which have become the most frequent cause of FA-related death. The Biologics License Application (BLA) for FA remains on track for submission to the U.S. Food and Drug Administration (FDA) in the first half of 2024. RP-L102 is an investigational gene therapy that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral (LV) vectors to contain a functional copy of the FANCA gene.

Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. These forward-looking statements include, but are not limited to, statements concerning Rocket's expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA, Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, Rocket's plans for the advancement of its DD program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies.

Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a new product candidates.