$RCKTROCKET PHARMACEUTICALS, INC.

Active trials

• phase2A Gene Therapy Study of RP-A501 in Male Patients With Danon Diseaserecruitingn=14
• phase2Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiencysuspendedn=10
• phase1A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)recruitingn=9
• —Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102enrolling by invitationn=14
• —Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapyactive not recruitingn=9
• —Danon Disease Natural History Studyrecruitingn=60
• phase1A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathyrecruitingn=8
• —BAG3-DCM Natural History Studynot yet recruitingn=30