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A father fights to create and then save his daughter’s gene therapy, the White House nears a decision on an FDA chief, and the Trump administration pushes drugmakers to reshore generics manufacturing.
A quest to save Grace — and help rare disease patients everywhere
With $70 million and seemingly every fiber of his being, Matt Wilsey built a gene therapy company from scratch — recruiting Nobel laureates and biotech veterans to will an experimental treatment into existence for his daughter Grace. The treatment, for the ultra-rare disorder NGLY1 deficiency, landed her back in the hospital before she slowly began to recover, STAT’s Jason Mast and Matt Herper write.
