Search the database for deliberate release of GM medicinal products

Displaying 1 - 2 of 2
EU record number Title Company / Sponsor Treated organism Genetic modification
B/BE/26/BVW3 A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long-term safety follow up part, in ambulant boys with Duchenne Muscular Dystrophy GENETHON Humans Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human dystrophin (hMD1) gene
B/BE/18/BVW7 Phase III trial investigating a AAV vector containing a variant of human factor IX gene administered to adult subjects with severe or moderately-severe hemophilia B uniQure biopharma B.V. Humans Recombinant adeno associated viral vector containing the wild type modified to express the Padua derivative of human coagulation factor IX cDNA

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