Very good no nonsense talk with Doug
Towards the end Doug rules out a basket trial on the LGMD variants. (I think - he mumbles it)... We'll start with (next year) a confirmatory trial on 9003 (LGMD2E). It's via an accelerated approval path, so the goal is to show the creation of the needed protein at 3 months after the one-time dose. So, it should be a quick trial. I'm guessing that it will then be followed by the same deal for the next LGMD variant drug, which is a combo - where the needed protein is introduced via half of it being in one capsid and the other half of the protein being delivered in a second capsid - simultaneously - and joining finally in the cell. That could be a separate trial for that variant and interesting approach, or it could be part of a larger "basket trial" with it and any other LGMD variant drugs that LRK has ready to go by then.