Gene Therapy, LYS-SAF302, Lowers Heparan Sulfate Levels, Trial Data Show

Gene Therapy, LYS-SAF302, Lowers Heparan Sulfate Levels, Trial Data Show

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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 the cerebrospinal fluid (CSF; the liquid surrounding the brain and spinal cord) and indicate a therapeutic potential for LYS-SAF302. The AAVance trial faced several setbacks last year. In June, the study was placed on hold after MRI revealed brain…

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