NCT06399107 - Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

August 1, 2024

Primary Completion Date

November 10, 2025

Study Completion Date

December 28, 2025

Conditions

Sickle Cell DiseaseSickle-Cell Disease With Crisis

Interventions

GENETIC

Drug Product is administered by IV infusion following myeloablative conditioning with busulfan

An autologous CD34+ cell-enriched population from patients with sickle cell disease (SCD), which includes hematopoietic stem cells (HSCs) that have been transduced with the BAH243 lentiviral vector (LVV) carrying the βA-T87Q-globin gene, is preserved in a cryopreservation solution.

Trial Locations (1)

086-373

RECRUITING

District One Hospital, Beijing