Ear­ly da­ta snap­shots of Ax­o­van­t's gene ther­a­pies for Parkin­son's, Tay Sachs ap­pear promis­ing

Fol­low­ing the col­lapse of its neu­ro-fo­cused strat­e­gy, Ax­o­vant’s for­ay in­to gene ther­a­pies — with their po­ten­tial for one-shot, long-term cures — has gen­er­at­ed con­sid­er­able in­ter­est. On Mon­day, the biotech pro­vid­ed an ear­ly pos­i­tive snap­shot of two of its pro­grams: in Parkin­son’s and in­fan­tile Tay-Sachs dis­ease.

Two pa­tients were giv­en the low­est dose of Ax­o­vant’s Parkin­son’s gene ther­a­py — AXO-Lenti-PD — as part of a mid-stage study called SUN­RISE-PD. Pa­tients were as­sessed three months af­ter their dose, hav­ing been washed out of their oral lev­odopa ther­a­py — the gold stan­dard treat­ment for Parkin­son’s. On a physi­cian-rat­ed scale (UP­DRS) as­sess­ing mo­tor func­tion (the Part III score), both pa­tients ex­pe­ri­enced an im­prove­ment. The scale mea­sures scores rang­ing from 0 to 108, with low­er scores in­di­cat­ing im­prove­ment. At three months, one pa­tient saw an im­prove­ment of 14 points, the oth­er 36 points — trans­lat­ing to an av­er­age im­prove­ment of 25 points or a mean 42% from base­line, Ax­o­vant said.

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