Preferred Label : Verbrinacogene Setparvovec;
NCIt definition : A synthetic, liver-directed adeno-associated virus (AAV) capsid AAVS3 encoding a variant
of human coagulation factor IX with a gain-of-function Padua mutation (R338L), that
may potentially be used to treat hemophilia B (congenital factor IX deficiency; Christmas
disease). Upon administration of verbrinacogene setparvovec 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 : KT8AC75KUX;
CAS number : 2364604-87-3;
Molecule name : FLT-180a;
NCI Metathesaurus CUI : CL1413088;
Codes from synonyms : 114506;
Origin ID : C175872;
UMLS CUI : C5446808;
Semantic type(s)
- concept_is_in_subset
