Rocket Pharmaceuticals Inc.

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

Role: lead

Danon Disease Natural History Study

Role: lead

A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

Role: lead

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Role: lead

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Role: lead

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

Role: lead

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Role: lead

BAG3-DCM Natural History Study

Role: lead

PKP2-ACM Natural History Study

Role: lead

Gene Therapy for Fanconi Anemia, Complementation Group A

Role: lead

Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

Role: lead

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Role: lead

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Role: lead

Natural History of Danon Disease

Role: lead

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Role: lead

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Role: lead

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

Role: lead

The Natural History of Danon Disease

Role: collaborator

A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

Role: lead

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

Role: lead