How one father built a biotech, Grace Science, to save his daughter

Matt Wilsey has dedicated the last ten years of his life and $70 million to find a cure for his daughter Grace, who suffers from a fatal, ultra-rare disease known as NGLY1 deficiency. This genetic condition has caused Grace to face significant developmental challenges, leaving her unable to speak or walk properly.

After treating 10 patients, including Grace, in a clinical trial conducted by his company, Wilsey finds himself in a challenging position. Despite the progress made, the company is now out of funds and lacks the necessary requirements for FDA approval. However, Wilsey remains undeterred and continues to press the agency, setting a crucial test case for regulators and others developing treatments for rare, deadly diseases.

The journey has not been easy for Wilsey and his family. Three weeks ago, Grace underwent a gene therapy designed to alleviate her symptoms and potentially save her life. Wilsey played a significant role in developing this therapy, hiring scientists, advisors, and investors to support the cause. His unwavering faith has been a source of strength throughout the process.

Despite coming to terms with the fact that Grace may never lead an independent life, Wilsey remains hopeful that the treatment will improve her quality of life. He believes that the strategies and efforts put into developing this therapy could pave the way for curing numerous rare diseases in the future.

Unfortunately, the therapy meant to save Grace’s life resulted in her deteriorating health, landing her back in the hospital. As Wilsey sat by her side, praying for her recovery, he knew that the stakes were high not only for his daughter but for the entire pharmaceutical industry watching closely.

The challenges faced by Wilsey and his company highlight the complexities of developing treatments for rare diseases. The story of Grace and her father serves as a poignant reminder of the resilience and determination required to push boundaries in medical research and bring hope to those in need.