Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Low Risk

Score: 30/100

Failure Rate

10.5%

2 terminated/withdrawn out of 19 trials

Success Rate

81.8%

-4.7% vs industry average

Late-Stage Pipeline

21%

4 trials in Phase 3/4

Results Transparency

89%

8 of 9 completed trials have results

Key Signals

2 recruiting8 with results

Enrollment Performance

Analytics

Phase 1

4(33.3%)

Phase 3

4(33.3%)

Phase 2

3(25.0%)

Early Phase 1

1(8.3%)

12Total

Phase 1(4)

Phase 3(4)

Phase 2(3)

Early Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (19)

Showing 19 of 19 trials

NCT05445128Phase 2Terminated

Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

Role: collaborator

NCT06224413Recruiting

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

Role: lead

NCT05353647Phase 2Active Not Recruiting

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

Role: collaborator

NCT02633943Active Not Recruiting

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Role: lead

NCT04628585Enrolling By Invitation

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

Role: lead

NCT02140554Phase 1Completed

A Study Evaluating the Safety and Efficacy of Lovo-cel in Severe Sickle Cell Disease

Role: lead

NCT02698579Active Not Recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Role: lead

NCT06271512Recruiting

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

Role: lead

NCT04413539Completed

Evaluation of Impact of Disease on Quality of Life, Education and Socio-professional Integration of Adults and Parents of Children Living with Sickle- Cell Disease in France

Role: collaborator

NCT04293185Phase 3Active Not Recruiting

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

Role: lead

NCT03852498Phase 3Completed

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Role: lead

NCT03131934Early Phase 1Active Not Recruiting

Immunotherapy With Tacrolimus Resistant EBV CTL for Lymphoproliferative Disease After Solid Organ Transplant

Role: collaborator

NCT03207009Phase 3Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Role: lead

NCT02906202Phase 3Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Role: lead

NCT02658929Phase 1Completed

Study of bb2121 in Multiple Myeloma

Role: collaborator

NCT02151526Phase 1Completed

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

Role: lead

NCT01896102Phase 2Completed

A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Role: lead

NCT02204904Terminated

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Role: lead

NCT01745120Phase 1Completed

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

Role: lead

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