Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field

There was no shortage of enthusiasm at the inaugural UC San Diego Gene Therapy Initiative Symposium hosted at the Roth Auditorium at the prestigious Sanford Consortium, where researchers, clinicians, academia, industry, and patient advocates shared discoveries in gene therapy, a modern-day treatment that's revolutionizing medicine.

CIRM was a platinum sponsor of the event.

The gene therapy field has experienced tremendous progress in recent years. In 2023, there were 1,894 active gene therapy clinical trials, and the first regulatory authorization of a CRISPR-based gene editing therapy in the world was approved by the FDA for sickle cell disease.

Simply put, a gene therapy is a technique that modifies a person's genes to treat or cure disease. Gene therapies can work in several ways. This may include replacing a disease-causing gene with a healthy copy of the gene, inactivating a disease-causing gene that is not functioning properly, or introducing a new or modified gene into the body to help treat a disease.

To date, the California Institute for Regenerative Medicine (CIRM) has awarded more than $1 billion to support gene therapy projects for a wide range of diseases and conditions. Despite this and other critical funding support, many challenges remain in paving the way for further advancements in gene therapy. The UC San Diego Gene Therapy Initiative and its inaugural symposium were a step forward in advancing these sought after medical breakthroughs through resource sharing, collaboration, and partnerships.

Collaborating to Fuel Gene Therapy Progress

Nancy Stack, who has witnessed firsthand the immense potential of gene therapy in her own family, was thrilled to see symposium attendees collaborating and exchanging knowledge. She and her husband Geoffrey "Jeff" Stack provided a $5 million gift which helped launch the Initiative. They both gave opening remarks at the event, welcoming the crowd of 200 attendees.

"The Gene Therapy Initiative at UC San Diego is one of the most exciting things happening in the world of medicine because we've already seen what happens with a gene therapy for our rare disease," Nancy said.

When their daughter Natalie was a baby, she was diagnosed with cystinosis, a genetic, metabolic defect which causes damage to the kidneys, liver, and other organs. Cystinosis impacts over 2,000 people worldwide and just over 600 people in the United States.

To fuel research for the condition, Nancy and Jeff founded the Cystinosis Research Foundationand provided support to UC San Diego over the years to fund cystinosis research. That research is being continued today by Stephanie Cherqui, PhD, professor of pediatrics at UC San Diego School of Medicine and chair of the Cystinosis Stem Cell and Gene Therapy Consortium.

With CIRM funding support, Cherqui developed a gene therapy approach to cystinosis. The therapy modifies a patient's own blood stem cells with a functional version of the defective CTNS gene. The Stack's daughter, Natalie, participated in the trial and had a significant decrease of the toxic buildup of cystine in her body.

Cherqui along with Alysson Muotri, PhD, a professor in the Departments of Pediatrics and Cellular and Molecular Medicine at UC San Diego School of Medicine, are leading the Gene Therapy Initiative.

"From a more global perspective I think we are joining a momentum where gene therapy is making several strides on conditions that have had no cure in the past and now offers opportunity and hope for people suffering from these conditions," Muotri said.

Highlighting Advancements in Gene Therapy Research

Muotri was one of many researchers at the UC San Diego Gene Therapy Initiative symposium putting the spotlight on how gene therapies are being investigated and utilized to treat and better understand rare diseases like Friedreich's ataxia, cystinosis, spinal muscular atrophy, Rett syndrome, and ALS.

He and Cherqui emphasized that while many gene therapies have focused on treating rare diseases, they have the potential to be applied for broader use and to treat more prevalent conditions like Alzheimer's and various neurological conditions.

In addition, patient advocates and institutions like CIRM and the National Institute of Health (NIH) highlighted the importance of partnerships in advancing gene therapies. CIRM's Lisa Kadyk, PhD, presented funding opportunities and an overview of CIRM's research portfolio, which spans numerous disease areas.

Throughout the event, researchers-including trainees in CIRM's education and training programs-presented research findings during a scientific poster session.

This knowledge and resource sharing is exactly what Jeff Stack hoped for and envisioned at the inaugural symposium.

"I think what we see at the symposium is ultimately what we had hoped would happen, which is this incredible group of people-extraordinarily bright, brilliant people here-learning about things, talking about collaboration, creating different synergies and working together," Jeff said. "And that's exactly what we had hoped to do when we funded the Gene Therapy Initiative, because it's so important to have the collaboration and the cooperation among scientists to get things accomplished."

The leadership at the UC San Diego Gene Therapy Initiative foresees a future where collaboration among various stakeholders in the field will accelerate the development and clinical application of new gene therapy treatments for devastating disorders. In this spirit, the UC San Diego Gene Therapy Initiative inaugural symposium sparked discussions on potential partnerships and future opportunities to advance gene therapy products from bench-to bedside.

Learn more about the UC San Diego Gene Therapy Initiative here.

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