Study Summary
This study is an open-label, non-randomized, single-dose Phase 1/2 trial involving around 85 adult and pediatric participants aged between 2 and 50 years with sickle cell disease (SCD). It aims to assess the effectiveness of hematopoietic stem cell transplantation (HSCT) using BAH243 for SCD.
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Drug Product is administered by IV infusion following myeloablative conditioning with busulfanGENETIC
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.
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| District One Hospital | Beijing | Beijing Municipality | China |