SAF-301

SAF-301 is an investigational gene therapy designed for the treatment of mucopolysaccharidosis type III A (MPS IIIA, Sanfilippo syndrome type A). The therapy employs adeno-associated viral vector serotype 10 (AAV10) to deliver functional copies of the human N-sulfoglucosamine sulfohydrolase (SGSH) and sulfatase modifying factor 1 (SUMF1) cDNAs directly into the brain, aiming to compensate for the genetic defects that result in heparan sulfate accumulation and neurodegeneration in patients with MPS IIIA. Intracerebral administration is achieved via neurosurgical procedures with direct brain injections. Developed and sponsored by Lysogene, SAF-301 has demonstrated safety and tolerability in initial clinical trials, with some stabilization in brain atrophy and modest improvements in patient behavior, attention, and sleep in a small cohort of pediatric patients.

Brand names

SAF-301SAF301SAF 301

Other names

LYS-SAF301LYS-SAF-301LYS-SAF 301