Etranacogene Dezaparvovec

Preferred Label : Etranacogene Dezaparvovec;

NCIt synonyms : Etranacogene Dezaparvovec-drlb;

NCIt definition : A non-replicating, recombinant adeno-associated virus serotype 5 (AAV5) encoding a variant of human coagulation factor IX with a gain-of-function Padua mutation (R338L), under control of a liver-specific promotor 1 (LP1), that may potentially be used to treat hemophilia B (congenital factor IX deficiency; Christmas disease). Upon administration of etranacogene dezaparvovec and transduction of hepatocytes, human coagulation factor IX is produced. This replaces missing factor IX and may normalize the activated partial thromboplastin time (aPTT), increase blood clotting, and reduce and control bleeding episodes.;

UNII : Z5XCD5Q9RL;

CAS number : 2156583-26-3;

Drug name : Hemgenix;

Molecule name : AMT-061;

NCI Metathesaurus CUI : CL1405981;

Codes from synonyms : 114383;

Details

Origin ID

C171798;

UMLS CUI

C5419304;

Semantic type(s)
- Nucleic Acid, Nucleoside, or Nucleotide [UMLS semantic type]
- Pharmacologic Substance [UMLS semantic type]
UMLS correspondences (same concept)
- etranacogene dezaparvovec [ATC Code]
concept_is_in_subset
- FDA Established Names and Unique Ingredient Identifier Codes Terminology [NCIt concept]
- HemOnc Agent Terminology [NCIt concept]

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