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BAH243
- Development stage
- Unknown
- Lead developer
- BioRay
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
BAH243 is an ex vivo gene therapy currently in clinical development for the treatment of sickle cell disease (SCD). The therapy utilizes a lentiviral vector to transduce a patient's own (autologous) hematopoietic stem and progenitor cells (HSPCs) with a functional globin gene. These modified cells are then re-infused into the patient following myeloablative conditioning, where they engraft in the bone marrow and produce red blood cells containing functional hemoglobin. This process aims to prevent the sickling of red blood cells, thereby reducing vaso-occlusive crises and other chronic complications associated with SCD. BAH243 is being evaluated in Phase 1/2 clinical trials to assess its safety and efficacy in both adult and pediatric populations.
- Other names
- BAH243 Lentiviral VectorBAH-243 Lentiviral VectorBAH 243 Lentiviral Vector
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