> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Audentes Therapeutics Regenerative Medicine Designation
- URL: https://www.fdaweb.com/audentes-therapeutics-regenerative-medicine-designation/
- Published: 2018-08-21T12:00:00.000Z
- Updated: 2026-09-15T00:27:04.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5142169

FDA has granted Audentes Therapeutics a regenerative medicine advanced therapy (RMAT) designation for AT132 and its use in treating X-linked myotubular myopathy. The RMAT designation was granted based on positive interim clinical data from ASPIRO, an ongoing Phase 1/2 clinical study. The data, according to the company, showed encouraging initial evidence of safety and efficacy in the first dose cohort, including significant improvements in neuromuscular function, increased respiratory function as demonstrated by reductions in ventilator dependence and gains in respiratory muscle strength. “In addition to these functional outcome measures, muscle biopsy results from the first three patients treated in the study at the 24-week timepoint demonstrate highly efficient tissue transduction as indicated by vector copy number, robust myotubularin protein expression as assessed by western blot, and significant improvement in histology,” it says.