Search the database for deliberate release of GM medicinal products
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| EU record number | Title | Company / Sponsor | Treated organism | Genetic modification |
|---|---|---|---|---|
| B/BE/25/BVW8 | A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy | Solid Biosciences Inc. | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human micro-dystrophin (h-µD5) gene |
| Only notified under the "contained use" procedure. Dossier submitted on . | A Phase I/II study of the safety and efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in subjects with severe sickle disease | CRISPR Therapeutic AG | Humans | Modified cells expressing Hemoglobin F (HbF) |