United States
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. is a late-stage biotechnology company dedicated to developing innovative gene therapies for rare and life-threatening diseases. Headquartered in Cranbury, New Jersey, the company operates through its subsidiaries in the United States.
Gene Therapy Programs
In Vivo Adeno-Associated Viral (AAV) Programs:
- RP-A501 (Danon Disease - DD): A Phase 2 trial targeting a multi-organ lysosomal disorder leading to early heart failure.
- RP-A601 (PKP2-ACM): A Phase 1 program for Plakophilin-2 Arrhythmogenic Cardiomyopathy, a severe inheritable cardiac disorder.
- BAG3-DCM: A preclinical-stage therapy for BAG3 Dilated Cardiomyopathy, characterized by progressive heart wall thinning.
In Vivo Lentiviral (LV) Programs:
- RP-L201 (LAD-I): Targets Leukocyte Adhesion Deficiency-I, a genetic immune system disorder.
- RP-L102 (Fanconi Anemia - FA): Addresses a bone marrow defect causing faulty blood cell production.
- RP-L301 (PKD): Focuses on Pyruvate Kinase Deficiency, a red blood cell disorder leading to chronic anemia.
Strategic Partnerships
Rocket Pharmaceuticals has established key license agreements with leading institutions, including:
- Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas
- UCL Business PLC
- The Regents of the University of California
- REGENXBIO, Inc.
- Temple University
Founded in 1999, the company continues to advance cutting-edge therapies to address unmet medical needs in rare diseases.
