CAS 2364604-87-3(CA)VERBRINACOGENE SETPARVOVEC(INNE)VERBRINACOGENE SETPARVOVEC(INNF)VERBRINACOGEN SETPARVOVEC
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(SLE)
VERBRINAKOGEN SETPARVOVEK(INNC)
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS OF A MODIFIED LIVER-TROPIC SEROTYPE (S3) (RAAV REP2- CAPS3) VECTOR, ENCODING A GAIN-OF-FUNCTION VARIANT (R338L; PADUA) OF CODON-OPTIMISED HUMAN COAGULATION FACTOR IX (HFIX) UNDER THE CONTROL OF A LIVERSPECIFIC PROMOTER (FRE1) COMPRISING A TRUNCATED VERSION OF THE HUMAN APOLIPOPROTEIN E LOCUS CONTROL REGION (I.E. ENHANCER) WITH A TRUNCATED VERSION OF THE HUMAN ALPHA-1-ANTITRYPSIN PROMOTER(WHO)