Latest results from MTM gene therapy trial

Published Date
Jenny Sharpe
AAV virus

Audentes Therapeutics has provided an update on its ‘ASPIRO’ gene therapy trial. The study is evaluating the safety and efficacy of AT132 in young children with X-linked myotubular myopathy (X-MTM).

Since releasing initial data from ASPIRO, the company has treated three more children with AT132. So far, six children have been treated and monitored for up to 24 weeks.

The majority of treated participants have show improvements in neuromuscular and respiratory function. One child is now able to stand without support and no longer needs to use a BiPAP ventilator.

Dr Suyash Prasad, Senior Vice President and Chief Medical Officer at Audentes, said in a press release:

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.

Whilst the efficacy data is promising, there have been a few safety concerns. An independent committee will now review all of the data and advise Audentes on testing higher doses (dose-escalation). The company expects to provide an update on its plans in the third quarter of 2018.

Further information

Read this press release and community letter from Audentes Therapeutics

Read our myotubular myopathy factsheet

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