IFEZUNTIRGENE INILPARVOVEC

UNII:	VB2ZC1P6P4
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	IFEZUNTIRGENE INILPARVOVEC
GSRS Full Record:	VB2ZC1P6P4 i

2407582-19-6
A RECOMBINANT, REPLICATION DEFICIENT ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING A MICRORNA (MIRNA) TARGETING THE EXON 1 OF HUMAN HUNTINGTIN PROTEIN (HTT) MRNA (MIHTT). THE EXPRESSION CASSETTE CONSISTS OF A CHIMERIC CHICKEN BETA-ACTIN PROMOTER, THE MIHTT SEQUENCE ENGINEERED IN THE MIR-451 BACKBONE, WITH THE UPSTREAM REGION OF MIR-451 CONTAINING AN INACTIVATED MIR-144 SEQUENCE, AND A HUMAN GROWTH HORMONE POLYADENYLATION SIGNAL, FLANKED BY AAV2 5' AND 3' INVERTED TERMINAL REPEATS (ITRS)
AAV5(765)-MIHTT
AMT-130
DNA (recombinant replication-deficient adeno-associated virus 5 vector AMT-130 chicken β-actin promoter plus human huntingtin mRNA-targeting microRNA miHTT-specifying)
IFEZUNTIRGENE INILPARVOVEC [USAN]
ifezuntirgene inilparvovec [INN]
rAAV5-miHTT

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.

IFEZUNTIRGENE INILPARVOVEC

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