The Guidance Arrived

Low Volume and Equity Mispricing

Everything predicted in March came true this morning, and the market doesn’t really care.

On March 24th, I wrote about a draft FDA guidance that had been sent to the White House for review, with the title “Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing.”

I told subscribers that this document was different in kind, not just degree — that it wasn’t about manufacturing standards or IND application requirements, but about epistemology itself, about whether prior natural history data, external controls, and cumulative real-world evidence can legitimately serve as the evidentiary backbone of a regulatory submission.

I said the bear case for AMT-130 rested on a single pillar: that the data cannot be evaluated without a randomized controlled trial. And I said that if this guidance were finalized, that pillar collapses — not because the clinical data changed, but because the regulatory standard would explicitly catch up to the scientific reality of rare disease gene therapy development.

This morning, June 2nd, 2026, the guidance published.