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

 /article Ref: PRNewswire | Image: Dennis McCain