About the Gene Therapy Initiative

Established in 2023 at UC San Diego, the Gene Therapy Initiative (GTI) was made possible by a visionary $5 million gift from the Nancy and Geoffrey Stack Foundation. The Stacks' daughter Natalie was diagnosed with cystinosis, inspiring their dedication to expanding treatment options for families facing rare genetic diseases. The initiative is focused on developing gene-based therapies: treatments that address diseases caused by missing or faulty genes.  

These conditions can affect several organ systems, and most have no cures — only symptom control, astronomical healthcare costs, and the risk of premature death. Many rare diseases affect small populations of fewer than 200,000 people in the U.S., which limits research opportunities and commercial incentive to develop treatments. Yet collectively, the more than 7,000 known rare diseases affect an estimated 350–400 million people worldwide, representing a massive health and economic burden and a profound unmet need.

Bridging the Gap: From Lab to Clinic

Beyond rare diseases, there is a global push to make gene therapies more widely accessible and to explore applications for common disorders. Yet many discoveries never leave the lab, held back by challenges in translating research into treatments, gaps in commercialization training, and limited industry collaboration. To bridge this gap, GTI supports early-stage gene therapy development through seed funding, translational research training, entrepreneurial mentorship, and a network connecting academia, biotech, and patient communities. Its leadership and advisors bring extensive experience advancing genetic therapies into clinical trials, helping accelerate breakthroughs that can change lives.