SAF-301 / Lysogene, Alcyone - LARVOL DELTA

An improved AAV vector for neurological correction of the mouse model of Mucopolysaccharidosis IIIA. (PubMed, Hum Gene Ther) - "Following preclinical evaluation in MPSIIIA mice, an adeno-associated virus vector of serotype rh10 designed to deliver SGSH and sulfatase modifying factor 1 (SAF301) was trialed in four MPSIIIA patients, showing good tolerance and absence of adverse events with some improvements in neurocognitive measures. Biodistribution of SAF302 was further assessed using GFP (SAF302GFP), indicating that vector spread was limited to the area around the injection tract. Further modification of the injection strategy to a single depth with higher injection volume increased vector distribution leading to more widespread GFP distribution and sustained expression, suggesting this approach should be adopted in future trials."

Journal • Preclinical