Audentes Reports Next Phase Development of AT132 for X-linked Myotubular Myopathy (XLMTM) with RMAT Designation

 Audentes Reports Next Phase Development of AT132 for X-linked Myotubular Myopathy (XLMTM) with RMAT Designation

Audentes Reports Next Phase Development of AT132 for X-linked Myotubular Myopathy (XLMTM) with RMAT Designation

Shots:

  • The update in the development is based on ASPIRO P-I/II study results assessing AT132 in 12 patients aged≤ 5 yrs. following a pre-IND meeting of AT132 with FDA in Dec, 2018 for XLMTM, occurred due to mutations in the MTM1 gene
  • The FDA meeting is in response to review nonclinical, clinical, chemistry, manufacturing and controls (CMC) data leading to submission of AT132’s BLA. The ASPIRO P-I/II study resulted in safe and effective muscle biopsy
  • AT132 includes an AAV8 vector containing a functional copy of the MTM1 gene, encode myotubularin protein. AT132 has received FDA’s RMAT, Rare Pediatric Disease, FT and ODD, and PRIME and Orphan Drug designations by the EMA. The preclinical development of AT132 was conducted with Genethon

Click here to read full press release/article | Ref: PRNewswire | Image: Dennis McCain

Shiwani Sharma

Shiwani Sharma was Senior Editor at PharmaShots. She has in-depth knowledge of the life sciences industry including the Pharma and Biotech sectors. Any articles written by her can be contacted at connect@pharmashots.com.

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