Mother leaning down touching foreheads with her son with CALD

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)
  • PH 2/3 
    Cerebral Adrenoleukodystrophy (Starbeam ALD-102)
  • PH 3 
    Cerebral Adrenoleukodystrophy (ALD-104)
betibeglogene autotemcel gene therapy (beti-cel; formerly LentiGlobinTM for β-thalassemia)
  • PH 3 
    APVD IN EU
    TDT Non-β0/β0 genotypes (Northstar-2, HGB-207)
  • PH 3 
    TDT β0/β0 genotypes (Northstar-3, HGB-212)
lovotibeglogene autotemcel gene therapy (lovo-cel; formerly LentiGlobinTM for sickle cell disease)
  • PH 3 

    SCD (HGB-210)

  • PH 1/2 

    SCD (HGB-206)

BCL11A shRNA (mir)1
  • PH 1 

    Sickle Cell Disease

MULTIPLE UNDISCLOSED

  • PRE-C 

    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.