Search the database for deliberate release of GM medicinal products
Displaying 1 - 3 of 3
| 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. |
| B/BE/25/BVW5 | GenePHIT: A study to learn more about how well a new gene therapy (AB-1002) works and its safety in participants with congestive heart failure | Asklepios BioPharmaceutical (AskBio) | Humans | Non-replicating recombinant vector derived from adeno-associated virus AAV2i8 carrying the transgene encoding for the human activated Inhibitor-1c ( I-1c) |
| B/BE/19/BVW2 | A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels Receiving Prophylactic FVIII | BioMarin Pharmaceutical Inc. | Humans | Recombinant adeno-associated virus serotype 5 (AAV5) containing a gene cassette expressing the human coagulation factor VIII (FVIII) gene |