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2026
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Another Step Closer to a Cure: A Statement from the Family of Pediatric Patient #2
October 26, 2026 | Cystinosis Research Foundation
The second pediatric patient has officially received the stem cell and gene therapy treatment at UC San Diego, marking a historic milestone not only for our foundation, but for the entire scientific community.
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UC San Diego & Rady Children’s Health Designated as NORD® Rare Disease Center of Excellence
September 21, 2026 | UC San Diego Today
The designation joins UC San Diego School of Medicine and Rady Children’s with a national network spanning more than 150 academic medical centers, research institutions and children’s hospitals working together to advance rare disease care and research in Southern California and nationwide.
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Farah Sheikh Receives National ISHR-NAS Innovator Award
June 17, 2026 | UC San Diego Today
Farah Sheikh, PhD, professor of medicine at the University of California San Diego School of Medicine, was recently honored by the International Society for Heart Research (ISHR) North American Section with the 2026 Innovator Award. She delivered the Innovator Award Lecture at the ISHR North American Section Annual Meeting in Minneapolis.
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Experimental Gene Therapy Shields Brain from Toxic Protein Damage
May 26, 2026 | UC San Diego Today
A harmful protein called TDP-43 plays a major role in brain diseases like frontotemporal dementia and is also found in many people with Alzheimer’s disease An experimental gene therapy developed by UC San Diego researchers helped protect brain cells from that damage
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Six UC San Diego Faculty Elected to National Academy of Inventors for 2026
February 26, 2026 | UC San Diego Today
Six professors from the University of California San Diego have been named Senior Members of the National Academy of Inventors (NAI) in recognition of their outstanding achievements in innovation. The UC San Diego honorees in NAI’s 2026 class of Senior Members are Omar Akbari, Brian P. Head (GTI Seed Grant Recipient), Abdoulaye Ndao, Kenneth Vecchio, Edward Wang and Yunde Zhao.
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UCSD Scientists Report Early Success With Cystinosis Gene Therapy in First-in-Human Trial
February 18, 2026 | Precision Medicine Online
Reporting early clinical results of a first-of-its-kind hematopoietic stem cell–based gene therapy for cystinosis, a rare inherited lysosomal storage disorder. In a Phase I/II trial at the University of California, San Diego, the investigational therapy demonstrated sustained reductions in cystine levels and stable organ function in most adult patients with advanced disease, supporting continued development and ongoing evaluation in pediatric patients.
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California Grants $7.4 Million to Advance Gene-Edited Stem Cell Therapy for Friedreich’s Ataxia
January 8, 2026 | UC San Diego Today
Funding supports safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients.
2025
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UC San Diego lab lands FDA approval for breakthrough clinical trials
December 31, 2025 | The San Diego Union-Tribune
Two projects explore the rare Pitt-Hopkins and Rett syndromes using organoids that mimic organs.
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From Wish to Reality — The Gene Therapy Initiative at UC San Diego
December 16, 2025 | UC San Diego Today
UC San Diego is a world leader in gene therapy research — one child’s wish helped them get there.
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MaddieBio: How a Father’s Love Drives Cutting-Edge Science
June 26, 2025 | UC San Diego Today
The GTI is built on the belief that collaboration drives impact. A recent feature from UC San Diego Today highlights this mission in action, sharing how a GTI-facilitated connection brought together biotech entrepreneur Gavenraj Singh Sodhi and GTI co-director Dr. Alysson Muotri. Their shared interests have evolved into an exploration of brain organoid technology as a potential pathway for identifying treatments for rare diseases, including those affecting Sodhi’s daughter, Maddie.
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Novartis CYStem Phase I/II Clinical Trial in Cystinosis Posted on ClinicalTrials.gov
April 2025 | Cystinosis Research Foundation
The pharmaceutical company Novartis has announced that the CYStem Phase I/II clinical trial for a pediatric population with cystinosis has been posted on ClinicalTrials.gov. This builds on the success of a CGT product that was first tested at UC San Diego in adults
Read About Trial
2024
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Inaugural UC San Diego Gene Therapy Initiative Symposium is fueling progress in the field
September 30, 2024 | The Stem Cellar
The inaugural symposium brought together experts, funders, and patient advocates to highlight cutting-edge gene therapy advances and the power of collaboration.
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GTI Leadership Shares Clinical and Translational Insights at 27th Annual ASGCT Meeting
May 2024
GTI leaders Chamindra Laverty, Alysson Muotri, and Stephanie Cherqui presented at the 27th Annual American Society for Gene and Cell Therapy meeting in Baltimore, Maryland, this May. Their talks highlighted key lessons in AAV gene therapy safety and efficacy, emerging applications of brain organoids in gene therapy, and the translational bench-to-bedside-to-bench cycle advancing ex vivo gene therapy -
RARE Drug Development Symposium
May 2024
Betty Cabrera, GTI Director of Research Engagement, participated as a panelist at the RARE Drug Development Symposium in Philadelphia, where discussions centered on innovative commercial and non-profit funding models for preclinical drug development. During the session, GTI showcased how UC San Diego researchers are leveraging rare disease research to drive advances in more prevalent disease.
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RARE Revolution Magazine - Issue NO 027
Spring 2024 | Rare Revolution
Rare Revolution: To bring about a dramatic and wide reaching change in conditions and attitudes for the rare disease community.
The Women in RARE edition highlights the women involved with working on rare diseases. Dr. Cherqui was interviewed about her journey in gene therapy, leading to the culmination of the Gene Therapy Initiative.
2023
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UC San Diego Chancellor’s Medal and Lifetime Legacy Award Recipients Announced
November 28, 2023 | UC San Diego Today
The University of California San Diego announced the recipients of the 2023 Chancellor's Medal and Lifetime Legacy Award. The recipients have all helped advanced health care and medicine in unique, impactful ways.
Nancy and Geoffrey Stack founded the Cystinosis Research Foundation in 2003. They also helped launch the UC San Diego Gene Therapy Initiative in 2023.
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SDBJ Business Women of the Year 2023 Finalists
November 20, 2023 | San Diego Business Journal
San Diego Business Journal delivers the market’s most complete package of business news and information on San Diego County’s companies, industries and business people. SDNJ covers commercial real estate, banking and finance, biotech, health care, technology, defense, tourism, manufacturing, professional services and more.Dr. Stephanie Cherqui was honored as a nominee for Woman of the Year 2023.
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UC San Diego Launches Gene Therapy Initiative
August 31, 2023 | RARE Daily
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. -
UC San Diego Launches Gene Therapy Initiative, Targeting Treatments for Rare Diseases
August 29, 2023 | UC San Diego Today
A $5 million gift from the Nancy and Geoffrey Stack Foundation has established the new initiative to find novel therapies for children and adults with genetic diseases. -
UCSD launches gene therapy initiative with help of $5 million gift from Stack foundation
August 29, 2023 | The San Diego Union-Tribune
A $5 million gift from Nancy and Geoffrey Stack will support the launch of UC San Diego's Gene Therapy Initiative. Nancy and Jeff have supported the research on cystinosis following their daughter's diagnosis. The Stacks hope that their gift will help further the future of gene therapy.