Re: Phenomenal Explanation of In-Vivo Gene Editing
>>> IMO in-vivo gene editing will work very well the second time around.
Yup. SM reiterated in several places what I mentioned after ER call, that the ROI for MPS is very low so there is no room for a third shot on the MPS goal. The second shot MUST be the best shot since there won’t be another. So they are throwing everything into this final try and coordinating the manufacturing transition needed for that with the handoff of Hemo A which likely frees up some equipment (low volume reactors) and people to be repurposed. Note that he mentioned it can take 11 months to grow a batch of AAVs. That certainly comports with what he’s giving us for the timing of the re-start of MPS trials.