Enthusiasm ran high at the inaugural UC San Diego Gene Therapy Initiative Symposium at the Roth Auditorium at the Sanford Consortium. Researchers, clinicians, and patient advocates gathered to share new discoveries in gene therapy—a rapidly evolving field that is reshaping modern medicine.
CIRM served as a platinum sponsor of the event.
Era of progress
The gene therapy field has experienced tremendous progress in recent years. In 2023, there were 1,894 active gene therapy clinical trials. The same year the FDA approved the first CRISPR-based gene editing therapy for sickle cell disease in the world.
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.
Progress in cystinosis
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 Foundation and 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. As a result she 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, are leading the Gene Therapy Initiative.
“From a more global perspective gene therapy is making strides on conditions that have had no cure in the past and now offers opportunity and hope,” Muotri said.
Highlighting Advancements in Gene Therapy Research
Muotri was one of several researchers at the UC San Diego Gene Therapy Initiative symposium highlighting how gene therapies are being developed. These include treatments and better understand rare diseases such as Friedreich’s ataxia, cystinosis, and ALA. He and Cherqui noted that while many gene therapies focus on rare diseases, these approaches could also be applied to more common conditions. That includes Alzheimer’s and other neurological disorders.
Patient advocates and institutions such as CIRM also stressed the importance of partnerships in advancing gene therapy. CIRM’s Lisa Kadyk, PhD, shared funding opportunities and an overview of CIRM’s broad research portfolio.
Researchers, including trainees from CIRM’s education and training programs, shared their findings during a scientific poster session. The exchange of knowledge and resources reflected exactly what Jeff Stack hoped to see at the inaugural symposium.
“What we’re seeing here is what we envisioned — an incredible group of extraordinarily bright people learning, discussing collaborations, building synergies, and working together,” Jeff said. “That’s why we funded the Gene Therapy Initiative. Collaboration is essential for scientists to get things done.”
• Leaders of the UC San Diego Gene Therapy Initiative envision stronger collaboration across the field to accelerate the development and clinical use of new gene therapies for serious disorders.
• The Initiative’s inaugural symposium encouraged these connections, sparking discussions about partnerships and future opportunities to advance gene therapy from bench to bedside.
Learn more about the UC San Diego Gene Therapy Initiative here.
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