Af­ter gene ther­a­py deaths, Astel­las to re­vis­it fa­tal neu­ro­mus­cu­lar con­di­tion in part­ner­ship with biotech start­up

Four years ago, a biotech com­pa­ny pre­sent­ed da­ta so pow­er­ful it made sci­en­tists cry.

Speak­ing to a packed room at an an­nu­al cell and gene ther­a­py con­fer­ence in 2019, Au­dentes Ther­a­peu­tics played a video of a young boy who could scarce­ly move and re­quired a ven­ti­la­tor to breathe. Born with a fa­tal neu­ro­mus­cu­lar con­di­tion called X-linked my­otubu­lar my­opa­thy, for which there are no treat­ments, he was out of op­tions un­til Au­dentes came along.

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