The Rome Cohort, page-79

I earlier raised the question - what would be the PUL 2.0 results once an optimal dose for ATL1102 is established and what would be the PUL 2.0 results in combination therapy using ATL1102 and an exon skipping or gene therapy drug?

In the meantime I've had the opportunity to listen to an interview with the Founder of the Jett Foundation who spoke of her son's experience in the eteplirsen trial for non-ambulatory Duchene patients back in 2015-16. She speaks of the subtlety of changes that you can't necessarily capture in a 6 minute walk test or a pulmonary function test but they are improvements that have given back a level of independence and enjoyment; activities that were important to them that they had lost prior to starting therapy. In the interview, eteplirsen is described as a foundational drug that produces dystrophin but importantly she says what can we add on top of that...maybe an anti-fibrotic, maybe an anti-inflammatory drug.

Fast forward to 2020. Indeed, what would be the PUL 2.0 results in combination therapy using ATL1102 and an exon skipping or gene therapy drug? What kind of difference could combination therapy make in the lives of Duchenne patients. Could we be standing on the edge of unexpected developments that will propel clinical progress forward.