SAN FRANCISCO — Matt Wilsey, founder of the biotech company Grace Science, is advocating for the U.S. Food and Drug Administration (FDA) to approve an experimental gene therapy for NGLY1 deficiency. This effort continues despite the FDA stating the company lacks necessary data for approval and reports of patient deterioration following treatment.

Wilsey established Grace Science after spending a decade working to develop a treatment for his daughter, Grace Wilsey, who has NGLY1 deficiency. This genetic condition is associated with profound developmental challenges. Wilsey invested $70 million in this development, hiring scientists and recruiting advisers, including Nobel Prize winners, to work on the therapy.

Grace Science conducted a clinical trial that treated 10 patients, including 15-year-old Grace Wilsey. Grace Wilsey received the gene therapy three weeks before July 9, 2026. Following the treatment, she was hospitalized, and her condition worsened.

The company, which has also brought together families of other children diagnosed with NGLY1 deficiency, has secured investors and donors. However, Grace Science is currently out of funds. Wilsey communicated with his staff, stating, "We carry the hopes of many. I'm not just talking about NGLY1 families. I receive emails, calls, and texts from professionals and other advocates. They are blown away by what we have accomplished and hope we are an ice breaker for them. Our trial has the potential to really boost / save a decimated field."

Why It Matters

This situation illustrates the challenges in bringing experimental treatments for rare genetic disorders to market, particularly when financial resources are depleted and regulatory requirements are unmet. The push for FDA approval by Grace Science, despite these operational and regulatory hurdles, indicates the pressure involved in developing therapies for conditions like NGLY1 deficiency, which can have profound developmental impacts on affected individuals.