Search the database for deliberate release of GM medicinal products
Displaying 1 - 3 of 3
| EU record number | Title | Company / Sponsor | Treated organism | Genetic modification |
|---|---|---|---|---|
| B/BE/25/BVW4 | An Open-Label Dose-Escalation Study to Assess the Safety and Tolerability of a Single Intravitreal Injection of SPVN20 Gene Therapy in Subjects with No Light Perception Due to End-Stage Rod-Cone Dystrophy, and Who Retain Dormant Foveal Cone Photoreceptors | SparingVision | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV2 carrying the G protein-gated inwardly rectifying potassium (GIRK) channel 1, mutated for an F137S amino-acid substitution (GIRK1(F137S)) gene |
| Only notified under the "contained use" procedure. Dossier submitted on 20/08/2018. | 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) |
| Only notified under the "contained use" procedure. Dossier submitted on 21/02/2018. | Efficacy and safety of bilateral intravitreal injection of GS010: a randomized, double-masked, placebo-controlled trial in subjects affected with G11778A ND4 leber hereditary optic neuropathy for up to one year. | GenSight Biologics | Humans | human wild-type MT-NDA (mitochondrial NADH Dehydrogenase 4) |