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CIRACIGÉN GOLPARVOVEC
RECOMBINANT, SELF-COMPLEMENTARY NON-REPLICATING ADENOASSOCIATED VIRUS VECTOR WITH OLIG-001* CAPSID (RAAV-OLIG-001) ENCODING CODON-OPTIMIZED HUMAN ASPARTOACYLASE (ASPA, ASP, ACY2) UNDER CONTROL OF A CYTOMEGALOVIRUS (CMV) ENHANCER/CHICKEN BETA-ACTIN (CBH) HYBRID PROMOTER AND TERMINATED BY A BOVINE GROWTH HORMONE POLYADENYLATION SIGNAL, FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS). THE VECTOR GENOME IS A HEAD-TO-HEAD, SELFCOMPLEMENTARY DIMER, WITH THE VECTOR GENOME CASSETTE LOCATED 5' OF THE MUTATED INTERNAL INVERTED TERMINAL REPEAT (DELTA-ITR) IN A REVERSE COMPLEMENTARY ORIENTATION AND 3' OF THE DELTA-ITR IN A FORWARD ORIENTATION. *THE OLIG-001 CAPSID IS A CHIMERIC MIXTURE OF AAV1, 2, 6, 8 AND 9 THAT FACILITATES ENTRY INTO OLIGODENDROCYTES