rebisufligene etisparvovec

Rebisufligene etisparvovec is an investigational in vivo gene therapy designed for the treatment of Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A. It uses a self-complementary adeno-associated virus serotype 9 (scAAV9) vector to deliver a functional copy of the human SGSH gene to cells, primarily targeting the central nervous system and peripheral organs. The therapy aims to restore sulfamidase enzyme activity, addressing the underlying deficiency that leads to abnormal accumulation of heparan sulfate and progressive neurodegeneration in affected patients. Rebisufligene etisparvovec is administered as a one-time intravenous infusion and has received multiple regulatory designations including Orphan Drug status in both the US and EU[1][3][6].

Other names

rebisufligene etisparvovec