About the Gene Therapy Initiative

Founded in 2023 at UC San Diego, GTI was launched with a visionary $5 million gift from the Nancy and Geoffrey Stack Foundation. The Stacks’ personal experience—through their daughter Natalie’s diagnosis with cystinosis—drives their commitment to expanding treatment options for families confronting rare genetic diseases.

The GTI is dedicated to advancing gene-based treatments for genetic diseases, which are caused by missing or mutated genes and can affect nearly every organ system, including the brain, heart, and nervous system. Many of these disorders are rare, impacting fewer than 200,000 people in the United States, and often lack effective treatments because limited patient populations restrict research opportunities.

There is also a world-wide effort to make gene therapies more accessible to patients and expand applications to more common diseases. However, much of the ingenuity needed to realize these goals never leaves the confines of academic laboratories. This can be attributed to obscure paths to translating discoveries to marketable therapies, lack of training in commercialization concepts, and missed opportunities to partner with industry.

The initiative fuels the early‑stage development of gene‑based therapies by providing seed funding, entrepreneurial mentorship, and a collaborative network that bridges academia, biotech, and patient communities. GTI’s leadership and advisors bring proven experience in developing genetic therapies that are currently in clinical trials for metabolic, cardiac, and other disorders, helping to accelerate medical breakthroughs.