Unnamed ARC Syndrome Program

The Unnamed ARC Syndrome Program is an ex-vivo lentiviral-based gene therapy being developed by Platelet Targeted Therapeutics for the treatment of Arthrogryposis, Renal dysfunction, and Cholestasis (ARC) Syndrome. ARC Syndrome is a rare, fatal multisystem disorder caused by mutations in the VPS33B or VPS16B genes, which encode proteins essential for intracellular vesicle trafficking and the biogenesis of platelet alpha-granules. This gene therapy approach involves the ex-vivo transduction of hematopoietic stem cells with a lentiviral vector carrying a functional copy of the deficient gene, aiming to restore normal cellular function and alleviate the severe clinical manifestations of the disease, including bleeding diathesis and organ dysfunction.

Other names

ARC Syndrome ProgramVPS33B/VPS16B gene therapyPlatelet Targeted Therapeutics-VPS33B-VPS16B-gene therapy-ARC Syndrome (Arthrogryposis, Renal dysfunction, Cholestasis)