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BIOgenetiX
United States
- Ownership
- Private
- Development stage
- Other
01
Overview
Genetix Biotherapeutics has FDA-approved gene therapies including LYFGENIA for sickle cell disease, ZYNTEGLO for β-thalassemia, and SKYSONA for early active cerebral adrenoleukodystrophy (CALD). These one-time administered therapies use the patient's own hematopoietic stem cells modified ex vivo to add functional genes, offering potential curative benefits based on decades of clinical data.[1][3][4]
- Therapeutic areas
- HematologyNeurology
- Modalities
- Gene therapy
- Industry
- Biotech
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