Search the database for deliberate release of GM medicinal products
| EU record number |
Title |
Company / Sponsor |
Treated organism |
Genetic modification |
| B/BE/25/BVW3 |
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B |
CSL Behring LLC |
Humans |
Recombinant adeno associated viral vector containing the codon-optimised human PADUA factor IX (FIX) 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) |
| Only notified under the "contained use" procedure. Dossier submitted on
. |
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) |
