Thirty to Sixty Days

A Question of What Counts as Evidence

A new draft FDA guidance was sent to the White House for review on March 23rd. The title: “Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing.”

Read that again.

Leveraging Prior Knowledge. In gene therapy development. Incorporating genome editing.

That is the evidentiary architecture of the entire AMT-130 program (Enroll-HD natural history data, external controls, prior clinical knowledge from an already-enrolled patient cohort) being potentially codified into formal FDA guidance. Coincidentally, it appears to be moving now, in the transitional window between Prasad’s departure and the normalization of CBER leadership, with career staff back at the wheel.

Here is what I know, and what it means for the thesis for AMT-130 and other related gene therapies.