Gene Therapy for Fanconi Anemia, Complementation Group A

Summary

The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

Timeline

Start

2020-07-15

Primary completion

2026-05-05

Completion

2026-05-05

Drugs

Indications