This article first appeared in The Information Point newsletter Our World in 2018.
Audentes Therapeutics recently released their first quarter 2018 results, in which they confirmed they had “Completed dosing of three additional patients as an expansion of the first dose cohort of ASPIRO, the Phase 1 / 2 clinical study of AT132, their gene therapy product.” They confirmed there were no significant adverse safety events in these three extra patients.
And during a presentation at the 21st annual meeting of the American Society of Gene and Cell Therapies in Chicago Dr. Suyash Prasad, Senior Vice President and Chief Medical Officer of Audentes stated “We continue to be highly encouraged by the profile of AT132 observed to date in the ASPIRO study. Patients treated in the initial cohort continue to make advancements in neuromuscular and respiratory function, highlighted by the fact that our earliest treated patient has now been ventilator independent for over eight weeks.”
Dr. Prasad continued, “We are pleased to see the encouraging trends in safety and efficacy continue in the recently enrolled Cohort 1 expansion patients, with early gains in CHOP-INTEND and MIP observed by the four-week timepoint and no significant treatment-related adverse events reported to date. We remain inspired by our patients and their families, and we look forward to continuing our work with the XLMTM community to advance this important work as rapidly as possible.”