Search the database for deliberate release of GM medicinal products
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 20 August 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 1 February 2005
.
A Controlled, Randomised, Parallel Group, Multicentre Study of the Efficacy and Safety of Herpes Simplex Virus-Thymidine Kinase Gene Therapy (CereproTM), with Subsequent Ganciclovir, for the Treatment of Patients with Operable High-Grade Glioma Ark Therapeutics Ltd
Humans
Thymidine Kinase (HSV-TK1)
