Dad Spends 10 Years Creating Gene Therapy for Daughter Grace

Matt Wilsey refused to accept that nothing could be done for his daughter Grace, who was diagnosed with NGLY1 deficiency, an ultra-rare genetic condition that prevented her from speaking or walking easily.

So he did something extraordinary. Over the past 10 years, Wilsey assembled a team of scientists, recruited Nobel Prize winners as advisers, and raised $70 million to develop a gene therapy from scratch.

The former tech executive had no medical background. But he had determination, faith, and a clear vision that his work could help not just Grace but hundreds of families facing similar devastating diagnoses.

Wilsey brought together families of other children with NGLY1 deficiency and built what he calls an "A-team" of investors and donors. His company has now treated 10 patients, including 15-year-old Grace, in clinical trials.

The journey hasn't been easy. Three weeks after receiving the experimental treatment, Grace ended up back in the hospital, weaker than before.

Wilsey sat beside his daughter, adjusting the plastic tube in her stomach, staying present in each 15-minute increment while his wife Kristen rested. He couldn't let himself think too far ahead or second-guess his choices.

The Ripple Effect

Wilsey's work has become a beacon for the rare disease community. He regularly receives emails, calls, and texts from advocates and professionals who see his efforts as potentially groundbreaking.

"We carry the hopes of many," he once wrote to his staff. "Our trial has the potential to really boost and save a decimated field."

His approach of assembling top scientific talent, building community support, and pushing forward despite regulatory hurdles is now being watched by the entire pharmaceutical industry. Other rare disease families see his work as an icebreaker that could pave the way for their own treatments.

While Wilsey's company has run out of money and still lacks full FDA approval, his decade-long quest demonstrates what's possible when a parent's love meets scientific innovation. His case is now setting a pivotal precedent for how regulators approach treatments for deadly rare diseases.

Through his devout Catholic faith and unwavering commitment, Wilsey has shown that even the rarest conditions don't have to mean giving up hope. He's created not just a potential treatment for his daughter, but a roadmap that could help save countless other children.

Grace may never live an independent life, but her father's work might help her live longer and perhaps even speak a few words. And in doing so, he's opened doors for families who once had nowhere to turn.