NIMATPAGENE PARIPARVOVEC

UNII:	C4J4L5V7XS
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	NIMATPAGENE PARIPARVOVEC
GSRS Full Record:	C4J4L5V7XS i

2773545-20-1
Capsomere, recombinant human adeno-associated virus AAV3B viral capsid protein VP1-VP2-VP3 complex (1:2:7), hexacontamer, complex with DNA (recombinant replication-defective adeno-associated virus serotype 3B vector VTX-801 liver-specific human α-1 antitrypsin promoter plus human 57-486-copper transporter ATP7B codon-optimized gene plus rabbit β-globin polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks) (1:1)
Nimatpagene Pariparvovec
Nimatpagene pariparvovec [WHO-DD]
Recombinant, non-replicating adeno-associated virus serotype 3B (rAAV3B) vector, encoding a shortened version of the human copper-transporting ATPase 2 (ATP7B) (also known as ATPase copper-transporting beta, Wilson disease-associated protein) which is devoid of the first four of the six metal binding sites (MBS) (mini-ATP7B), under control of a liver specific human alpha-1 antitrypsin (AAT) promoter and a synthetic polyadenylation sequence derived from the polyadenylation signal of the rabbit ß-globin gene, and flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs)
VTX-801
nimatpagene pariparvovec [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.

NIMATPAGENE PARIPARVOVEC

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