Sponsor pipeline

Genethon clinical trial pipeline

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Data updated daily from ClinicalTrials.gov, last refresh 2026-10-16

What changed in the last 30 days

First postedNew trial filingPhaseStatus
2026-10-02 NCT07856329 A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in… Phase 2/3 Recruiting

Active trials by phase

Phase 1/2
1
Phase 2
1
Phase 2/3
1
Not charted: 3 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials

NCT IDTitleConditionStatusUpdated
Phase 2/3
NCT07856329 A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in… Duchene Muscular Dystrophy Recruiting 2026-10-13
Phase 2
NCT06518005 Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe… Crigler-Najjar Syndrome Recruiting 2024-12-03
Phase 1/2
NCT02333760 Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott… Wiskott-Aldrich Syndrome Active, Not Recruiting 2021-06-03
Not applicable
NCT03466463 Gene Therapy for Severe Crigler Najjar Syndrome Crigler-Najjar Syndrome Recruiting 2023-03-28
NCT07633951 Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and… Neuromuscular Deficits Not Yet Recruiting 2026-06-08
Not specified
NCT03882827 Natural History of Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy Recruiting 2026-02-12

Disease areas by active trials

Duchenne Muscular Dystrophy (2)

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