dirloctocogene samoparvovec

Dirloctocogene samoparvovec is an investigational gene therapy developed for the treatment of hemophilia A (Factor VIII deficiency). It consists of an adeno-associated virus (AAV) vector—specifically, a bioengineered capsid derived from AAV3 (subtype LK03)—that delivers a codon-optimized cDNA encoding human coagulation factor VIII to liver cells. The therapy is administered as a one-time intravenous infusion and aims to enable patients' own livers to produce functional factor VIII, thereby reducing or eliminating the need for exogenous factor VIII replacement and decreasing bleeding episodes. Dirloctocogene samoparvovec has shown durable expression of FVIII and significant reductions in annualized bleeding rates in clinical trials. It is being developed by Spark Therapeutics, now part of Roche, and has received orphan drug designation in both the US and Europe as well as breakthrough therapy designation in the US for hemophilia A[1][2][4][5][6].

Other names

dirloctocogene samoparvovec