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ZAFTUCLENEGENE PIRUPARVOVEC

Chemical FDA Substance Registry (openFDA)
Chemical Identification
CAS Number2567881-78-9
Known Aliases (3)
Common Name
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR TSHA-118 CHICKEN .BETA.-ACTIN HYBRID PROMOTER PLUS HUMAN ACYL PROTEIN THIOESTER HYDROLASE PPT1 PRECURSOR TRANSGENE PLUS BOVINE GROWTH HORMONE POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TERMINAL REPEAT FLANKS-SPECIFYING) RECOMBINANT, SELF-COMPLEMENTARY (DIMERIC), NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (SCAAV9) VECTOR, ENCODING CODON-OPTIMISED HUMAN PALMITOYL-PROTEIN THIOESTERASE 1 (PPT1, CLN1), UNDER THE CONTROL OF A SYNTHETIC PROMOTER CONSISTING OF ACYTOMEGALOVIRUS (CMV) ENHANCER/CHICKEN .BETA.-ACTIN (CBA) PROMOTER AND A HYBRID INTRON DERIVED FROM CHICKEN .BETA.-ACTIN AND MINUTE VIRUS OF MICE (MVM) INTRONS, FOLLOWED BY A KOZAK SEQUENCE, AND TERMINATED BY A BOVINE GROWTH HORMONE (BGH) POLYADENYLATION (POLYA) SIGNAL AND THE VECTOR GENOME CONTAINS A MUTATED ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) INVERTED TERMINAL REPEAT (MITR) AND IS FLANKED BY AAV2 WILD-TYPE INVERTED TERMINAL REPEATS (WTITRS) zaftuclenegene piruparvovec [INN]
Data Sources (1)
Source Source ID Name in Source Properties
FDA Substance Registry (openFDA) R5X46L79RV ZAFTUCLENEGENE PIRUPARVOVEC CAS: 2567881-78-9
INN: 12046
SMS_ID: 300000031611
FDA UNII: R5X46L79RV
NCI_THESAURUS: C188620
Record Info
IDe5vNo2G6Mc887xhcL8MWrA
CreatedApril 10, 2026, 9:07 p.m.
ModifiedApril 10, 2026, 11:03 p.m.
Data SourceFDA Substance Registry (openFDA)