| Recruiting | A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 D Dilated Cardiomyopathy (DCM) | Phase 1 | 2026-06-01 |
| Not Yet Recruiting | BAG3-DCM Natural History Study Dilated Cardiomyopathy (DCM), Cardiovascular Diseases, Heart Diseases | — | 2026-05-01 |
| Suspended | Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency Pyruvate Kinase Deficiency | Phase 2 | 2026-04-01 |
| Recruiting | PKP2-ACM Natural History Study Cardiomyopathies, Heart Diseases, Cardiovascular Diseases | — | 2026-03-01 |
| Recruiting | Danon Disease Natural History Study Danon Disease | — | 2023-12-20 |
| Recruiting | A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease Danon Disease | Phase 2 | 2023-09-05 |
| Recruiting | A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopat PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Phase 1 | 2023-08-29 |
| Completed | Natural History of Danon Disease Danon Disease | — | 2022-09-26 |
| Active Not Recruiting | Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) Leukocyte Adhesion Deficiency | — | 2022-03-09 |
| Enrolling By Invitation | Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 Fanconi Anemia | — | 2022-02-01 |
| Terminated | A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis Infantile Malignant Osteopetrosis | Phase 1 | 2020-11-19 |
| Active Not Recruiting | Gene Therapy for Fanconi Anemia, Complementation Group A Fanconi Anemia Complementation Group A | Phase 2 | 2020-07-15 |
| Completed | Gene Therapy for Pyruvate Kinase Deficiency (PKD) Pyruvate Kinase Deficiency | Phase 1 | 2020-07-06 |
| Active Not Recruiting | Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy Fanconi Anemia Complementation Group A, Fanconi Anemia | — | 2020-06-01 |
| Active Not Recruiting | Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A Fanconi Anemia Complementation Group A | Phase 2 | 2019-11-28 |
| Completed | A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency Leukocyte Adhesion Defect - Type I | Phase 1 / Phase 2 | 2019-08-30 |
| Unknown | Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B Danon Disease | Phase 1 | 2019-04-17 |
| Withdrawn | A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Defici Leukocyte Adhesion Defect - Type I | Phase 1 | 2019-04-15 |
| Unknown | A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A Fanconi Anemia Complementation Group A | Phase 1 | 2019-01-11 |