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
. |
EXACT 2 is a randomized, multicenter, double-blind, placebo-controlled Phase 2b trial designed to evaluate the safety, feasibility, and efficacy of percutaneous endovascular catheter-facilitated intramyocardial administration of XC001 in patients with ref |
XyloCor Therapeutics |
Humans |
VEGF |
| 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 |
