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PASEMNOGÉN GLEPARVOVEC
PASEMNOGENE GLÉPARVOVEC
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (AAV9) VECTOR, ENCODING A CODON-OPTIMISED HUMAN SURVIVAL OF MOTOR NEURON 1 (SMN1) GENE, UNDER CONTROL OF THE CYTOMEGALOVIRUS (CMV) ENHANCER/PROMOTER AND TERMINATED BY A HUMAN GROWTH HORMONE POLYADENYLATION SIGNAL, FLANKED BY AAV2 INVERTED TERMINAL REPEATS. A HUMAN BETA-GLOBIN INTRON HAS BEEN INSERTED BETWEEN THE PROMOTER AND THE SMN1 CODING REGION. THE VIRUS VECTOR ALSO CONTAINS MUTATIONS IN THE VIRAL CAPSID PROTEIN VP1 (F422W AND Y446F)