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/BVW9 Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002 VectorY Therapeutics B.V. Humans Non-replicating recombinant AAV vector with a AAV5.2 capsid and carrying the transgene corresponding to the single-chain variable fragment that binds to human TDP-43
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)

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