ARTICLE | Clinical News

Audentes reports interim Phase I/II data for XLMTM candidate

January 5, 2018 9:50 PM UTC

Gene therapy company Audentes Therapeutics Inc. (NASDAQ:BOLD) reported interim data from the first cohort of the Phase I/II ASPIRO trial of AT132 to treat X-linked myotubular myopathy, a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death.

In a nine-month old patient, a 1x1014 vector genomes per kg (vg/kg) dose of AT132 improved neuromuscular function as measured by Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score from 29 points at baseline to 56 points at week 12. AT132 also improved respiratory function as measured by maximal inspiratory pressure (MIP) from 33 cm H2O at baseline to 80 cm H2O at week 12...

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Audentes Therapeutics Inc.

BCIQ Target Profiles

Myotubularin 1 (MTM1)