Key Highlights

Risk & Performance

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

0.0%

0 terminated/withdrawn out of 14 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

14%

2 trials in Phase 3/4

Results Transparency

43%

3 of 7 completed trials have results

Key Signals

3 with results

Enrollment Performance

Analytics

Phase 1

8(72.7%)

Phase 3

2(18.2%)

Phase 2

1(9.1%)

11Total

Phase 1(8)

Phase 3(2)

Phase 2(1)

Activity Timeline

Global Presence

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Clinical Trials (14)

Showing 14 of 14 trials

NCT03392987Phase 2Completed

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Role: lead

NCT06149403Phase 3Active Not Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

Role: lead

NCT03488394Phase 1Active Not Recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

Role: lead

NCT01560182Phase 1Completed

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Role: lead

NCT05368038Enrolling By Invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Role: collaborator

NCT04283227Phase 3Active Not Recruiting

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

Role: lead

NCT05687474Completed

Baby Detect : Genomic Newborn Screening

Role: collaborator

NCT04201405Phase 1Active Not Recruiting

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Role: collaborator

NCT05354219Unknown

Validation and Reliability of Iris Cameras in Mucopolysaccharidoses

Role: collaborator

NCT03765632Phase 1Completed

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Role: collaborator

NCT01852071Phase 1Completed

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

Role: collaborator

NCT02999984Phase 1Completed

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Role: collaborator

NCT01380990Phase 1Completed

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Role: collaborator

NCT02453477Phase 1Unknown

Gene Therapy for Transfusion Dependent Beta-thalassemia

Role: collaborator

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