OZUNACOGENE PARVEC

UNII:	EJU2EQH9H4
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	OZUNACOGENE PARVEC
GSRS Full Record:	EJU2EQH9H4 i

3062583-60-9
Ozunacogene Parvec
ozunacogene parvec [INN]
recombinant non-replicating bidirectional adeno-associated virus serotype 8 (rAAV8) vector encoding two copies of human coagulation factor IX (FIX, F9) in opposite tail-to-tail orientation. Each FIX transgene is preceded by a murine albumin splice acceptor to ensure DNA insertion into intron 1 of the albumin (ALB) locus when administered in association with taziguran and ziclumeran. One FIX transgene is terminated with a simian virus 40 (SV40) polyadenylation signal and one with a bovine growth hormone polyadenylation signal. Both transgenes are CpG depleted and do not encode the endogenous FIX signal peptide. The vector genome is flanked by AAV2 inverted terminal repeats (ITRs). Following factor IX (F9) DNA insertion into the ALB locus, the transcribed messenger RNA codes for a hybrid protein which includes the ALB signal peptide and F9 pro-peptide sequences that are both removed during the process of F9 protein maturation, resulting in wild-type human F9 protein

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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.

OZUNACOGENE PARVEC

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