Gene Therapy, LYS-SAF302, Lowers Heparan Sulfate Levels, Trial Data Show
All nine Sanfilippo type A patients analyzed to date in the ongoing Phase 2/3 clinical trial of the gene therapy LYS-SAF302 show reductions in the levels of heparan sulfate (HS), Lysogene, the therapy’s developer, reported. These reductions were specifically observed in…