Genetix leverages our expertise in genetic medicine to bring transformative treatments to patients in need
| Product | Indication | Research | Clinical development | Approved |
|---|---|---|---|---|
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LYFGENIA™ Sickle Cell Disease |
Sickle cell disease patients 12 years and older with a history of vaso-occlusive events |
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| Sickle cell disease patients under the age of 12 |
Ongoing Study: |
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ZYNTEGLO™ Transfusion-dependent |
Adult and pediatric patients with transfusion dependent beta-thalassemia |
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SKYSONA™ Cerebral Adrenoleukodystrophy |
Pediatric boys with early, active CALD without an available HLA-matched donor for allogenic HSCT* |
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CALD = cerebral adrenoleukodystrophy
*Human leukocyte antigen-matched donor for allogenic hematopoietic stem cell transplantation.
Product
Indication
Sickle cell disease patients 12 years and older with a history of vaso-occlusive events
Indication
Sickle cell disease patients under the age of 12
Ongoing Study: HGB-210
Product
ZYNTEGLO™
(begibeglogene autotemcel)
Transfusion-dependent
Beta-thalassemia
Indication
Adult and pediatric patients with transfusion dependent beta-thalassemia
Product
SKYSONA™
(elivaldogene autotemcel)
Cerebral Adrenoleukodystrophy
Indication
Pediatric boys with early, active CALD without an available HLA-matched donor for allogenic HSCT*
CALD = cerebral adrenoleukodystrophy
*Human leukocyte antigen-matched donor for allogenic hematopoietic stem cell transplantation.
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