Father spends $70 million on gene therapy trial for daughter with ultra-rare disease, faces FDA hurdles

Matt Wilsey has spent a decade and about $70 million trying to develop a gene therapy for his 15-year-old daughter Grace, who suffers from a fatal ultra-rare disease. His personal quest has turned into a small biotech effort that has enrolled ten patients, including Grace, in an early-stage clinical trial.

The trial has delivered a one-time gene therapy infusion to each patient, but the company does not yet have the data the FDA typically requires for approval. Funding has run out, leaving the program without the resources to complete the necessary studies.

Undeterred, Wilsey is urging the FDA to consider the therapy based on the limited data available, positioning the case as a pivotal test for regulators handling life-threatening rare diseases.

The situation underscores the broader challenges of bringing gene therapies for ultra-rare conditions to market, where high development costs and small patient numbers often clash with regulatory expectations.