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AVALOTCAGENE ONTAPARVOVEC

UNII:20E4R0BF12
Formula:
Preferred Substance Name:AVALOTCAGENE ONTAPARVOVEC
GSRS Full Record:

  • 2227000-30-6
  • A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/8 (RAAV REP2-CAP8) VECTOR, ENCODING CODON-OPTIMIZED, WILD-TYPE HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) UNDER THE CONTROL OF A HYBRID HUMAN THYROXINE-BINDING GLOBULIN (TBG) / HUMAN ALPHA 1-MICROGLOBULIN/BIKUNIN PRECURSOR (AMBP) PROMOTER/ENHANCER. THE VECTOR GENOME IS A HEAD-TO-HEAD DIMER, WITH THE VECTOR GENOME CASSETTE LOCATED 5' OF THE MUTATED INTERNAL INVERTED TERMINAL REPEAT (ITR) IN A REVERSE ORIENTATION AND THE VECTOR GENOME CASSETTE LOCATED 3' OF THE MUTATED INTERNAL ITR IN A FORWARD ORIENTATION
  • AVALOTCAGENE ONTAPARVOVEC [INN]
  • AVALOTCAGENE ONTAPARVOVEC [USAN]
  • DTX301
  • DTX-301
  • DTX-301 (ORNITHINE-TRANSCARBAMYLASE-GENE-THERAPY)
  • KFB12DX3PF
  • SCAAV8OTC



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