Search the database for deliberate release of GM medicinal products
EU record number
Title
Company / Sponsor
Treated organism
Genetic modification
B/BE/21/BVW3
Phase I/II study to evaluate the safety and effects on Progranulin levels of PR006 in patients with Fronto-Temporal Dementia with Progranulin mutations (FTD-GRN).
Prevail Therapeutics
Humans
The study involves a replication-incompetent adeno-associated virus, AAV9, lacking all AAV viral genes and encoding for the human Progranulin (GRN) 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 24 October 1996
.
Prospective, open-label, parallel-group, randomized, multicenter trial comparing the efficacy of surgery, radiation, and injection of murine cells producing herpes simplex thymidine kinase vector followed by intravenous ganciclovir against the efficacy of
Genetic therapy, Inc., Sandoz Pharma, Ltd
Humans
Thymidine Kinase (HSV-TK1), neomycin resistance (NeoR)