Lysogene (Paris:LYS) (FR0013233475 – LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869) presented at the WORLDSymposium™ 2021 by Dr. Michaël Hocquemiller, Head of Non-Clinical Development, during the Late-Breaking Science session held on Friday, February 12, 2021.
Concentration of heparan sulfate (HS) in the cerebrospinal fluid (CSF) has been analyzed in 9 patients so far, with samples at baseline (n=9), 6 months (n=8) and 12 months (n=5) after LYS-SAF302 administration. The concentration of abnormally elevated HS-derived oligosaccharides in the CSF was reduced from baseline by 27% and 30% at 6 and 12 months, respectively (p<0.005 and p<0.05 by paired t-test vs baseline, respectively).
