ZAFTUCLENEGENE PIRUPARVOVEC

UNII:	R5X46L79RV
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	ZAFTUCLENEGENE PIRUPARVOVEC
GSRS Full Record:	R5X46L79RV i

2567881-78-9
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]

Note

UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

ZAFTUCLENEGENE PIRUPARVOVEC

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