I thought one of the main reasons they chose to focus on non-ambulant population was the increased presence of T cells with the CD49d protein on the surface, ATL1102’s target (bio marker) to reduce inflammation.
Whilst being effective across the whole DMD population would be advantageous as far as increasing the total addressable market, imo we don’t want to spread the trial wings too far with the risk of muddying the results, let alone increasing trial cost out of our budget range. Not that we can change it now anyway.
Would be interested to get the thoughts of the more medically qualified. It’s no point targeting DMD boys who don’t have a significant immune response that our drug targets.
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