“AT132” Audentes Therapeutics treatment for myotubular myopathy

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):

https://lnkd.in/gn8GSFtq

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…