BioCentury
ARTICLE | Clinical News

Audentes gains on XLMTM readout

January 4, 2018 10:28 PM UTC

Gene therapy company Audentes Therapeutics Inc. (NASDAQ:BOLD) added $5.28 (15%) to $40.81 on Thursday after reporting 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. The company added almost $160 million to its market cap on the news.

In a nine-month old patient, a 1x10^14 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...

BCIQ Company Profiles

Audentes Therapeutics Inc.

BCIQ Target Profiles

Myotubularin 1 (MTM1)