Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

Summary

This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A. The study will evaluate gene therapy by transplantation of autologous CD34+ hematopoietic stem cells transduced ex vivo with the CD68-ET3 lentiviral vector.

Timeline

Start

2024-09-01

Primary completion

2029-08

Completion

2039-08

Drugs

Indications