Freeline’s early Gaucher disease data; Shorla raises $35M; Regenxbio to start pivotal DMD trial next year

Free­line’s ear­ly da­ta on gene ther­a­py for Gauch­er dis­ease: Free­line Ther­a­peu­tics said Wednes­day that its gene ther­a­py for Gauch­er dis­ease looked safe so far in two pa­tients who were dosed 13 and six weeks ago in a Phase I/II clin­i­cal tri­al. Gauch­er is a rare ge­net­ic dis­or­der that oc­curs when cer­tain fat­ty sub­stances build up in or­gans be­cause peo­ple don’t make enough of an en­zyme called GCase. The ther­a­py, called FLT201, al­so led to in­creas­es in GCase ac­tiv­i­ty in both pa­tients, Free­line said. The biotech’s stock $FRLN rose 10% Wednes­day morn­ing. — Lei Lei Wu

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