
let's recode the science
pipeline
Product Candidates
Severe Genetic Diseases
- Pre-Clinical
- Phase 1
- Phase 2
- Phase 3
elivaldogene autotemcel gene therapy (eli-cel, Lenti-DTM)
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Cerebral Adrenoleukodystrophy (Starbeam ALD-102)
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Cerebral Adrenoleukodystrophy (ALD-104)
betibeglogene autotemcel gene therapy (beti-cel; formerly LentiGlobinTM for β-thalassemia)
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TDT Non-β0/β0 genotypes (Northstar-2, HGB-207)
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TDT β0/β0 genotypes (Northstar-3, HGB-212)
lovotibeglogene autotemcel gene therapy (lovo-cel; formerly LentiGlobinTM for sickle cell disease)
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SCD (HGB-210)
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SCD (HGB-206)
BCL11A shRNA (mir)1
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Sickle Cell Disease
MULTIPLE UNDISCLOSED
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Undisclosed
Clinical Trials
To learn more about our clinical studies of investigational gene therapies in transfusion-dependent β-thalassemia, severe sickle cell disease, and cerebral adrenoleukodystrophy, visit our clinical trials page.