Search the database for deliberate release of GM medicinal products
| EU record number |
Title |
Company / Sponsor |
Treated organism |
Genetic modification |
| B/BE/26/BVW4 |
A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion, and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects with RS1-Associated X-linked Retinoschisis (LIGHTHOUSE) |
ASTENA Therapeutics |
Humans |
Non-replicating recombinant vector derived from adeno-associated virus AAV44.9 carrying functional copies of the human retinoschisin (hRS1) gene. |
| Only notified under the "contained use" procedure. Dossier submitted on
. |
A single-arm, open-label, multi-centre, phase I/II study evaluating the safety and clinical activity of QEL-001, an autologous CAR T regulatory cell treatment targeting HLA-A2, in HLA-A2/ A28neg patients that have received an HLA-A2pos liver transplant |
Quell Therapeutics Limited |
Humans |
BCMA CAR; shRNA CD3ζ; tCD34 |
| B/BE/18/BVW6 |
Global study of a recombinant adeno-associated virus designed to address the monogenic root cause of Spinal Muscular Atrophy |
AveXis |
Humans |
Recombinant adeno-associated virus serotype 9 (AAV9) containing the cDNA of the human SMN (Survival Motor Neuron) gene |
