XLMTM Gene Therapy: Q3 2018 Update from Audentes

Dr. Suyash Prasad, Audentes Chief Medical Officer, addressing the 2017 MTM-CNM Family Conference in Nashville

Yesterday, Audentes Therapeutics provided a quarterly company update, including information on their lead program - clinical development of AT132, a gene therapy product targeting X-Linked Myotubular Myopathy (XLMTM).  Their Phase 1/2 clinical trial, called "ASPIRO", is well underway, and they state:

"...we have recently completed enrollment of the Cohort 2 dose of 3x1014 vg/kg, including three treated patients and one untreated control patient.  We look forward to engaging with the FDA and EMA in the coming weeks to discuss development plans and the potential registration pathway for AT132, and to advance our goal of making this important treatment available to patients living with XLMTM as rapidly as possible."
​
Additional information in the full press release may be found on the Audentes website here.  Also, as has been their practice, Audentes has shared a letter with our community, which can be read here.
​

---