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DALNACOGENE PONPARVOVEC

UNII:XHE4MDR8C6
Type:INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:DALNACOGENE PONPARVOVEC
GSRS Full Record:
XHE4MDR8C6
i

  • 2771332-93-3
  • BBM-H901 Injection
  • Capsomere, recombinant liver-tropic adeno-associated virus AAV843 viral capsid protein VP1, complex with viral capsid protein VP2, complex with viral capsid protein VP3 decamer, hexacontamer, complex with DNA (recombinant replication-defective adeno-associated virus 2 vector recombinant liver-specific promoter Lxp-2.1 plus human DNA-dependent RNA polymerase II gene intron 1 plus human blood-coagulation factor IX Padua codon optimized gene 5′-region plus human immunoglobulin heavy chain variable region gene intron 2 plus human blood-coagulation factor IX Padua codon-optimized gene 3′-region plus recombinant mini polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying)
  • DALNACOGENE PONPARVOVEC [USAN]
  • DNA (recombinant replication-defective adeno-associated virus 2 vector recombinant liver-specific promoter Lxp2.1 plus human DNA-dependent RNA polymerase II gene intron 1 plus human blood-coagulation factor IX Padua codon-optimized gene 5′-region plus human immunoglobulin heavy chain variable region gene intron 2 plus human blood-coagulation factor IX Padua codon-optimized gene 3′-region plus recombinant mini polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying)
  • Dalnacogene Ponparvovec
  • dalnacogene ponparvovec [INN]
  • rAAV843 vector
  • recombinant, self-complementary, non-replicating adenoassociated virus, synthetic liver-tropic serotype 843 (rAAV843) vector, encoding a codon-optimised (CpG reduced) gain of function variant (Padua variant; R338L) of human coagulation factor IX (FIX), under control of a synthetic liver-specific Lxp2.1 promoter and a synthetic polyadenylation (polyA) sequence derived from the rabbit beta-globin gene polyA signal. The expression cassette also contains a human DNA-dependent RNA polymerase II gene intron, a Kozak sequence, a human immunoglobulin heavy chain variable region gene intron inserted into the middle of the FIX coding region, and an enhancer derived from the promoter region of human alpha-1-antitrypsin. The transgene is flanked by adenoassociated virus 2 (AAV2) inverted terminal repeats (ITRs)




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