BioCentury
ARTICLE | Clinical News

Audentes reports Phase I/II muscle biopsy data for myopathy gene therapy

August 10, 2018 6:26 PM UTC

Audentes Therapeutics Inc. (NASDAQ:BOLD) reported data from its 24-week muscle biopsy analyses of the first dose cohort and said an IDMC recommended proceeding to the second dose cohort of the Phase I/II ASPIRO trial evaluating AT132 in patients with X-linked myotubular myopathy (XLMTM). AT132 is an adeno-associated viral serotype 8 (AAV8) vector delivering the myotubularin 1 (MTM1) gene.

Among three evaluable patients in the open-label, dose-escalation, U.S. trial, a 1x1014 vector genomes per kg (vg/kg) dose of AT132 showed "highly efficient" tissue transduction as measured by increased vector copy per diploid genome from below the limit of detection at baseline to 6.2, 7.1 and 2.7, respectively, at week 24. Additionally, MTM1 protein expression as measured by Western blot increased from below the limit of detection at baseline in two patients to about 120% and 80% of normal at week 24, respectively, and from 5% at baseline to about 250% of normal in the third patient at week 24...

BCIQ Company Profiles

Audentes Therapeutics Inc.

BCIQ Target Profiles

Myotubularin 1 (MTM1)