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Product Candidates

Severe Genetic Diseases

Lenti-D™ Drug Product

  • Cerebral Adrenoleukodystrophy (Starbeam ALD-102): Phase 2/3
  • Cerebral Adrenoleukodystrophy (ALD-104): Phase 3

LentiGlobin™ Drug Product For β-Thalassemia

  • Transfusion-Dependent β-Thalassemia Non-β00 (HGB-207): Phase 2/3
  • Transfusion-Dependent β-Thalassemia β00 (HGB-212): Phase 2/3
  • Transfusion-Dependent β-Thalassemia (HGB-204): Phase 1/2
  • Transfusion-Dependent β-Thalassemia (HGB-205): Phase 1/2

LentiGlobin™ Drug Product For Sickle Cell Disease

  • Planned: Sickle Cell Disease (HGB-210): Phase 2/3
  • Sickle Cell Disease (HGB-206): Phase 1/2
  • Sickle Cell Disease (HGB-205): Phase 1/2

BCL11a shRNA (miR)

  • (Development is led by Dana-Farber/Boston Children’s Cancer and Blood Disorders Center)

    Sickle Cell Disease: Phase 1/2

Multiple Undisclosed

  • Hurler Syndrome (MPSI): Preclinical
  • Undisclosed: Preclinical


idecabtagene vicleucel (bb2121)

  • In Planning: Multiple Myeloma First Line: Phase 2/3
  • KarMMa-2: Multiple Myeloma Second Line (1 Prior): Phase 2/3
  • KarMMa-3: Multiple Myeloma Third Line (2-4 Prior): Phase 2/3
  • KarMMa: Multiple Myeloma ≥3 Prior Lines: Phase 2/3
  • CRB-401: Multiple Myeloma ≥3 Prior Lines: Phase 1/2


  • CRB-402: Multiple Myeloma ≥3 Prior Lines: Phase 1/2


  • (Development is led by Fred Hutch Cancer Research Institute)
    Merkel Cell Carcinoma: Phase 1/2

UNC CAR Collaboration

  • Solid Tumors: Preclinical


  • MAGE-A4 + Solid Tumors: Preclinical


  • DLBCL: Preclinical

DARIC Multi-Target

  • (Development is led by Seattle Children's Research Institute)
    AML: Preclinical

Multiple Undisclosed

  • Undisclosed: Preclinical

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.