RP-L102

BIOLOGICAL4 trials

Sponsors

Rocket Pharmaceuticals Inc.

Conditions

Fanconi AnemiaFanconi Anemia Complementation Group A

Phase 1

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

Rocket Pharmaceuticals Inc.Fanconi Anemia Complementation Group A

Start: 2019-01-11End: 2022-03-31Target: 2Updated: 2020-11-24

Phase 2

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

Active, not recruitingNCT04069533

Rocket Pharmaceuticals Inc.Fanconi Anemia Complementation Group A

Start: 2019-11-28End: 2025-02-28Updated: 2024-05-22

Gene Therapy for Fanconi Anemia, Complementation Group A

Active, not recruitingNCT04248439

Rocket Pharmaceuticals Inc.Fanconi Anemia Complementation Group A

Start: 2020-07-15End: 2026-05-05Target: 5Updated: 2025-12-22

Unknown Phase

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

Enrolling by invitationNCT07527975

Rocket Pharmaceuticals Inc.Fanconi Anemia

Start: 2022-02-01End: 2038-05-01Target: 14Updated: 2026-04-14

Related Papers

Non-Genotoxic Restoration of the Hematolymphoid System in Fanconi Anemia Mice through Antibody-Mediated Hematopoietic Stem Cell Transplantation

Blood2022-11-15

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-VivoFANCA Gene Insertion in Human Stem and Progenitor Cells

Blood2019-11-131 citations