Meet Our Leadership Team

Stephanie Cherqui, PhD

Director of Gene Therapy Initiative

Dr. Stephanie Cherqui, Professor of Pediatrics, is a trailblazer in gene‑therapy research, directing a laboratory that engineers hematopoietic stem cells to correct multi‑systemic degenerative diseases. Her pioneering work launched the first‑in‑human autologous stem‑cell gene therapy trial for cystinosis and was subsequently acquired by Novartis for $87.5 million to advance the program. Leveraging that success, Dr. Cherqui has secured more than $12 million in CIRM funding to translate gene‑edited therapies for Friedreich’s ataxia and is co-founder of Papillon Therapeutics. Recognized on BIOS’s 2023 “Beaker List” of Top Women in Academic Entrepreneurship and honored with the Rady Children’s Hospital Scientist Award of Excellence, she guides UC San Diego’s Gene Therapy Initiative with visionary leadership and a proven record of turning breakthroughs into viable treatments.

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Alysson Muotri, PhD

Co-Director of Gene Therapy Initiative

Dr. Alysson Muotri, Professor of Pediatrics, is a visionary leader in harnessing stem‑cell‑derived brain organoids to decode human neurodevelopment and devise gene‑therapy solutions for monogenic disorders such as Rett, Pitt‑Hopkins, and CDKL5 deficiency. He created the first human cellular model of Rett syndrome in 2010 and, in 2025, this led to IND clearance by the FDA for a novel gene‑therapy that precisely modulates gene expression in Pitt‑Hopkins syndrome without harmful over‑expression. A recipient of the NIH Director’s New Innovator award, Dr. Muotri also translates his discoveries into impact‑driven ventures, founding Tismoo—a biotech startup delivering personalized autism therapeutics. As co‑director of the Gene Therapy Initiative, he blends cutting‑edge science and entrepreneurial acumen to accelerate the path from organoid discovery to clinical reality.

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Betty Cabrera, MPH

Director of Engagement and Operations  

Betty Cabrera brings more than a decade of cell and gene‑therapy expertise to the UC San Diego Gene Therapy Initiative, where she leads a program that translates discovery‑stage science into IND‑enabling preclinical studies and strategic partnerships with biotech companies. Prior to joining UC San Diego, she directed operations for one of California’s first CIRM‑funded clinical‑research units, shepherding multiple IND‑enabling projects from the bench to early‑phase trials. Her hands‑on experience with clinical studies and patient registries gives her a nuanced understanding of the scientific, regulatory, and personal challenges faced by families affected by rare genetic diseases.