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Kiran Musunuru, MD, PhD, MPH, ML, MRA
Professor for Translational Research | University of Pennsylvania
Kiran Musunuru, MD, PhD, MPH, ML, MRA, is the Barry J. Gertz Professor for Translational Research at the Perelman School of Medicine and Co-Director of the Penn Medicine/CHOP Orphan Disease Center. A practicing cardiologist and dedicated educator, his research focuses on developing gene-editing therapies for cardiovascular and metabolic diseases. Dr. Musunuru also serves as Editor-in-Chief of The American Journal of Human Genetics. In 2025 he co-developed a breakthrough, personalized CRISPR therapy for an infant with a rare metabolic disease. He was named among the 100 Most Influential People of 2026 by TIME magazine.
2026 Speakers
Keynote Speaker
Featured Speakers
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Stephanie Cherqui, PhD
Professor of Pediatrics, Division of Genetics & Director of the UC San Diego Gene Therapy Initiative | UC San Diego
Stephanie Cherqui, Ph.D., is a Professor in the Department of Pediatrics, Division of Genetics at the University of California San Diego and Director of the UC San Diego Gene Therapy Initiative. Her research focused on developing hematopoietic stem cell gene therapy for metabolic and neurological disorders, with a distinguished record of translating fundamental discoveries into clinical applications. Among her most significant achievements is the development of the first-in-human autologous hematopoietic stem cell gene therapy for cystinosis, an inherited metabolic disorder. She is the Chair of the Cystinosis Stem Cell and Gene Therapy Consortium, and a member of the ASCGT Gene and Cell Therapy of Genetic and Metabolic Diseases committee. -
Brian P. Head, PhD
Professor of Anesthesiology & VA Research Career Scientist | UC San Diego & VA San Diego Healthcare System
Brian Head, PhD, is a Research Career Scientist at the VA San Diego Healthcare System and Professor of Anesthesiology at the University of California San Diego. He has over two decades of experience in research focused on AAV-mediated gene therapies for neurodegenerative diseases, including Alzheimer’s disease, amyotrophic lateral sclerosis, and TDP-43–related disorders. He was elected a Senior Member of the National Academy of Inventors in 2026. A gene therapy program he helped develop for ALS and that was acquired by Eikonoklastes Therapeutics received Investigational New Drug clearance from the U.S. Food and Drug Administration in 2025. -
Olivia Kim-McManus, MD
Associate Clinical Professor of Neurosciences | UC San Diego, Rady Children's Institute for Genomic Medicine
Olivia Kim-McManus, MD, is an Associate Clinical Professor of Neurosciences and a physician-scientist supported by the California Institute for Regenerative Medicine and the National Center for Advancing Translational Sciences. Board-certified in pediatric neurology, epilepsy, and clinical neurophysiology, her research focuses on precision therapeutics for rare genetic diseases, including antisense oligonucleotides. Dr. Kim-McManus has extensive IND regulatory experience and serves as principal investigator on multiple intrathecal and intraventricular clinical programs. She is also Vice Chief of the Neurology MSEC Section at Rady Children's Hospital and serves on several advisory and scientific review boards. -
Laurence Mignon, Ph.D
Vice President of Clinical Development | n-Lorem Foundation
Laurence Mignon, Ph.D., is Vice President of Clinical Development at n-Lorem Foundation. She has over 20 years of experience in academic research and clinical development focusing on rare diseases. She previously worked at Ionis Pharmaceuticals in Translational Medicine and Clinical Development, contributing to the development of Spinraza for spinal muscular atrophy and initiated the first clinical study in myotonic dystrophy type 1. She also held roles at Orexigen Therapeutics and the Neuroscience Education Institute. Dr. Mignon earned her Ph.D. in Pharmacology and Experimental Therapeutics from Loyola University Chicago and conducted neurology research at University of California Los Angeles. -
Alexis C. Komor, PhD
Associate Professor of Chemistry and Biochemistry | UC San Diego
Alexis Komor, PhD, is an Associate Professor in the Department of Chemistry and Biochemistry at the University of California San Diego. She received her BS in Chemistry from University of California Berkeley and her PhD from California Institute of Technology, and completed postdoctoral training with David Liu at Harvard University, where she co-developed base editing, a genome editing technology that avoids double-stranded DNA breaks. Her research focuses on advancing precision genome editing technologies, understanding their mechanisms, and applying them to study disease-associated mutations. -
Prashant Mali, PhD
Professor of Bioengineering | UC San Diego
Prashant Mali, Ph.D., is a Professor of Bioengineering at the University of California San Diego. His research lies at the intersection of synthetic biology and regenerative medicine, with a focus on developing gene- and cell-based therapeutic technologies. Dr. Mali earned his B.Tech. and M.Tech. degrees in Electrical Engineering from the Indian Institute of Technology Bombay, his Ph.D. in Biomedical Engineering from Johns Hopkins University, and completed postdoctoral training in Genetics at Harvard Medical School. He has received numerous honors and was elected to the American Institute for Medical and Biological Engineering in 2022 and the National Academy of Inventors in 2023.
Featured Panelists
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Dan Oliver, MBA
Co-founder & CEO | Rejuvenate Bio
Daniel Oliver is the CEO and co-founder of Rejuvenate Bio, a startup out of George Church’s Lab pioneering gene therapy solutions for age-related chronic diseases. Rejuvenate Bio’s initial focus on cardiovascular health involves a groundbreaking "one-shot" treatment for heart disease and obesity. The company’s validation in companion animals has led to partnerships with major players in animal health. Prior to Rejuvenate Bio, Daniel was awarded a Blavatnik Fellowship and he co-founded Voxel8, named one of the 50 most innovative companies by the MIT Technology Review. Daniel received his M.B.A. from Harvard Business School and has degrees in Mechanical Engineering and Business from the California Institute of Technology.
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Nicole Paulk, PhD
CEO & Founder | Siren Biotechnology
Dr. Nicole Paulk is the CEO and Founder of Siren Biotechnology and has dedicated her career to advancing gene therapy. Nicole is a pioneer in the development of next-generation AAV platforms for gene transfer and gene editing, directed evolution for engineered capsids, and multi-omic approaches to interrogate AAV biology for countless diseases. Before founding Siren, Nicole held various positions in academia and industry, and most notably was faculty in the UCSF Department of Biochemistry & Biophysics. Nicole has a B.S. in Microbiology, a Ph.D. in Gene Therapy from OHSU, and completed her Postdoctoral Fellowship and Instructorship in Human Gene Therapy at Stanford University before starting her lab at UCSF.
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Sharif Tabebordbar, PhD
Global Head of Cell and Gene Therapies | Novartis
Sharif Tabebordbar, PhD, is Global Head of Cell and Gene Therapies at the Novartis Biomedical Research. He earned his PhD in developmental and regenerative biology from Harvard University and completed postdoctoral training at the Broad Institute. Previously, he was Co-Founder and Chief Scientific Officer of Kate Therapeutics, which was acquired by Novartis in 2024. His research focuses on engineering AAV capsids and regulatory elements using machine learning to develop gene therapies for genetic diseases. He provided the first proof-of-concept for in vivo gene editing to treat Duchenne muscular dystrophy and developed key platforms for muscle-directed gene delivery.