PEBOCTOCOGENE CAMAPARVOVEC

UNII:	CFE96HL6SK
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	PEBOCTOCOGENE CAMAPARVOVEC
GSRS Full Record:	CFE96HL6SK i

2408734-39-2
A RECOMBINANT, NON-REPLICATING, ADENO-ASSOCIATED VIRUS SEROTYPE HU37 (AAVHU37) VECTOR, ENCODING A CODON-OPTIMISED B-DOMAIN DELETED SQ VARIANT OF HUMAN BLOOD-COAGULATION FACTOR VIII (HFVIII-SQ), UNDER THE CONTROL OF A HYBRID LIVER PROMOTER (MUS MUSCULUS TRANSTHYRETIN ENHANCER-HUMAN TRANSTHYRETIN PROMOTER) AND A SYNTHETIC POLYA SIGNAL SEQUENCE, FLANKED BY AAV2 INVERTED TERMINAL REPEATS; THE A2 AND A3 DOMAINS OF HFVIII ARE LINKED BY A DNA SEQUENCE ENCODING A 14-AMINO ACID PEPTIDE (SQ) FROM THE B DOMAIN
DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS HU37 VECTOR DTX201 MUS MUSCULUS TRANSTHYRETIN ENHANCER-HUMAN TRANSTHYRETIN PROMOTER PLUS HUMAN (1-764)-(1659-2351)-BLOOD-COAGULATION FACTOR VIII-SPECIFYING)
peboctocogene camaparvovec [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.

PEBOCTOCOGENE CAMAPARVOVEC

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