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
LENTI-DTM DRUG PRODUCT
  • PH 2/3 
    Cerebral Adrenoleukodystrophy (Starbeam ALD-102)
  • PH 2/3 
    Cerebral Adrenoleukodystrophy (ALD-104)
LENTIGLOBINTM PRODUCT CANDIDATE FOR TRANSFUSION-DEPENDENT β‑THALASSEMIA (TDT)
  • 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 PRODUCT CANDIDATE FOR SICKLE CELL DISEASE (SCD)
  • PH 2/3 

    SCD (HGB-210)

  • PH 1/2 

    SCD (HGB-206)

BCL11A shRNA (mir)1
  • PH 1 

    Sickle Cell Disease

MPSI GENE THERAPY APPROACH
  • PRE-C 

    Hurler Syndrome

MULTIPLE UNDISCLOSED

  • PRE-C 

    Undisclosed

Oncology
  • Pre-Clinical
  • Phase 1
  • Phase 2
  • Phase 3
IDE-CEL (BB2121)2
  • PH 1 

    Multiple Myeloma First Line (KarMMa-4)

  • PH 2 

    Multiple Myeloma Second Line (KarMMa-2)

  • PH 3 

    Multiple Myeloma Third Line (KarMMa-3)

  • PH 2 

    Multiple Myeloma Fourth Line+ (KarMMa)

  • PH 1 

    CRB-401: Multiple Myeloma ≥3 Prior Lines

BB212172
  • PH 1 

    CRB-402: Multiple Myeloma ≥3 Prior Lines

MCC1 TCR3
  • PH 1 

    Merkel Cell Carcinoma

UNC ONCOLOGY TARGET CAR4

  • PRE-C 

    Solid Tumors

MAGE-A4 TCR
  • PRE-C 

    MAGE A4 Positive Solid Tumors

DUAL B-CELL CAR
  • PRE-C 

    Diffuse Large B-Cell Lymphoma

DARIC MULTI-TARGET5

  • PRE-C 

    Acute Myeloid Leukemia

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.