Genethon
16
3
4
8
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
High Risk
Score: 60/100
12.5%
2 terminated/withdrawn out of 16 trials
80.0%
-6.5% vs industry average
0%
0 trials in Phase 3/4
0%
0 of 8 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (16)
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD)
Role: lead
Gene Therapy for X-linked Chronic Granulomatous Disease
Role: lead
Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I
Role: lead
Natural History of Duchenne Muscular Dystrophy
Role: lead
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Role: collaborator
Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
Role: lead
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
Role: collaborator
Gene Therapy for Severe Crigler Najjar Syndrome
Role: lead
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Role: lead
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Role: lead
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
Role: collaborator
Gene Therapy for WAS
Role: lead
Immunity Against AAV in Crigler Najjar Patient
Role: lead
Research of Biomarkers in Duchenne Muscular Dystrophy Patients
Role: lead
Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
Role: lead
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
Role: lead
All 16 trials loaded