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 
    APVD IN EU
    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)
LentiGlobinTM gene therapy for sickle cell disease (bb1111)
  • 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

Oncology
  • Pre-Clinical
  • Phase 1
  • Phase 2
  • Phase 3
idecabtagene vicleucel (ide-cel; bb2121)2
  • PH 2 

    KarMMa-2: Multiple Myeloma Second Line (1 Prior)

  • PH 3 

    KarMMa-3: Multiple Myeloma Third Line (2-4 Prior)

  • PH 1 

    KarMMa-4: Multiple Myeloma First Line

BB212172
  • PH 1 

    CRB-402: Multiple Myeloma ≥3 Prior Lines

NG MM CAR T

  • PRE-C 

    Solid NextGen CAR T - 4 approaches

MCC1 TCR3
  • PH 1 

    Merkel Cell Carcinoma

bbT369
  • PRE-C 

    bNHL

DARIC33
  • PRE-C 

    Pediatric AML

DARIC NextGEN

  • PRE-C 

    Adult AML

MAGE-A4 TCR4
  • PRE-C 

    MAGE-A4+ve Solid Tumors

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, cerebral adrenoleukodystrophy, and relapsed/refractory multiple myeloma, visit our clinical trials page.