Father invests $70 million in gene therapy quest for daughter with ultra‑rare NGLY1 deficiency, faces FDA scrutiny

Matt Wilsey has spent a decade and roughly $70 million trying to develop a gene therapy for his daughter Grace, who suffers from NGLY1 deficiency, an ultra‑rare genetic disorder.

NGLY1 deficiency is a fatal condition that causes severe developmental problems and currently has no approved treatment.

Wilsey’s effort has attracted leading scientists, Nobel laureates, and a network of investors, all working to create a therapy that could extend Grace’s life.

The next hurdle is convincing the Food and Drug Administration to approve the treatment, a step that could set precedents for the broader field of rare‑disease gene therapies.