MSB 2023 - awaiting for Ryoncil approval, page-57

I hope the new year sees you well LED but I personally think the real question is still can MSB have presented enough data.

I think the real questions are still the science and statistics ones. They are harder questions for most to answer but science and statistics do constrain what is possible and they aren't impossible for most people to learn.

How can MSB have a potency assay that's a reliable predictor of future product potency without measuring INF-gamma levels (as well as TNFR1 (or TNF alpha) in the batches?

How can CBER (the FDA) be sure that the good results from the 54 patient trial and the good survival data for four years (all of which come from only 3 donors - which can't supply enough for a commercial product themselves they have only so much bone marrow and will themselves be aging -so new donors will be needed - so potency assays that work will be needed) approve without seeing convincing data on such potency assays?

I can't see MSB retrospectively (after the 54 patient trial has already been done) getting enough predictive data on potency assays (which seem to need to be broader as TNFR1 doesn't pick up INF-gamma levels) collecting enough data - so I conclude more data has to be generated with potency assays beyond TNFR1 used in advance.

But we will see. Its just it might be quite a while until we do see as there isn't a time constraint (well not a publicly available one anyway - they have some internal guidelines apparently) on how long the FDA can take to reply to an IND update.

When they do reply I suppose MSB will report their reply if they deem it material (so they deem they have to report to update the market) but I wonder if they may not submit yet further IND updates still rather than make a formal BLA resubmission which carries some risk of rejection whereas IND updates don't.