UC San Diego Launches Gene Therapy Initiative

Rare Daily Staff

The University of California San Diego, backed with a $5 million gift from the Nancy and Geoffrey Stack Foundation, said it has established the UC San Diego Gene Therapy Initiative to develop treatments for rare, genetic diseases.

The Stacks’ daughter Natalie was less than a year old when she was diagnosed with cystinosis, a genetic defect which causes damage to the kidneys, liver, and other organs. At the time, the diagnosis was considered terminal.

To help their daughter, the couple established the Cystinosis Research Foundation. They also provided significant support to UC San Diego over the years to fund cystinosis research.

Stephanie Cherqui, professor of pediatrics at UC San Diego School of Medicine and chair of the Cystinosis Stem Cell and Gene Therapy Consortium, will co-direct the new UC San Diego Gene Therapy Initiative. Alysson Muotri, a professor in the departments of Pediatrics and Cellular and Molecular Medicine at UC San Diego School of Medicine, will also co-direct. The initiative will bring together key investigators, clinicians, knowledge and resources to foster new discoveries and chart the path forward for progress in gene therapy.

With the support from the Stacks, UC San Diego researchers were able to develop a slow-release version of a medication to help manage cystinosis, enabling patients to take the dosage every 12 hours, rather than every 6 hours. More recently, Cherqui developed a stem cell gene therapy approach to cystinosis, which has benefited patients including Natalie, who, at 32, has a career in social work and is planning her wedding in the fall.

“Our hope is that this opens the door to more effective therapies for those dealing with rare disorders and diseases,” said Nancy Stack. “We believe that the new Gene Therapy Initiative will give others hope, just like we received hope for our daughter thanks to new and better treatments.”