Richard Wilson, Astellas SVP, primary focus lead – genetic regulation

Fol­low­ing clin­i­cal hold lift, Astel­las posts ear­ly Pompe gene ther­a­py da­ta

Fresh out of a clin­i­cal hold, Astel­las is spot­light­ing da­ta on its gene ther­a­py for Pompe dis­ease.

In four pa­tients who got an in­fu­sion of the gene ther­a­py, called AT845, three dis­con­tin­ued en­zyme re­place­ment ther­a­py. As of the Sept. 15 cut­off, those three pa­tients have been off en­zyme re­place­ment ther­a­py for 19, 44 and 51 weeks, re­spec­tive­ly.

Astel­las did not bring any da­ta on the pri­ma­ry ef­fi­ca­cy end­point — en­zyme ex­pres­sion and ac­tiv­i­ty — though it did present sec­ondary ef­fi­ca­cy da­ta. The three par­tic­i­pants who were tak­en off ERT main­tained sim­i­lar re­sults in forced vi­tal ca­pac­i­ty, a mea­sure of lung ca­pac­i­ty, af­ter stop­ping bi­week­ly treat­ments. In ad­di­tion, two of the three had con­sis­tent re­sults on a six-minute walk test af­ter be­ing tak­en off ERT. The third saw a de­crease in dis­tance walked on the test af­ter they de­vel­oped a case of pe­riph­er­al neu­ropa­thy.

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