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UCL Biochemical Engineering



“AT132” Audentes Therapeutics treatment for myotubular myopathy

By Samir Nuseibeh, on 15 September 2021

Irrespective of the advances made in gene therapy over the past two decades, it is clear that we still don’t have a full grasp over the safety elements associated with them – particularly when high doses are involved.

This was sad news to arrive in my inbox today regarding the development of “AT132” by Audentes Therapeutics to treat myotubular myopathy (MTM1):


Trial: https://lnkd.in/gB9qr334

Transduction of muscle tissue is notoriously difficult and requires high viral genome doses in order to achieve sufficient expression of a given transgene. Evidently, the developers selected extremely high doses off the back of this demand and are now suffering the consequences of this choice.

Whilst I admire the developer’s commitment to helping the MTM1 community, it does beg the question – are high viral genome doses really an acceptable rationale for gene therapy development, considering the risks associated with safety? I suppose we’ll let the biopharmaceutical industry dictate that…

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