2026 Speakers

Keynote Speaker

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.

Featured Speakers

Mohsin Ahmed, MD, PhD
Clinical Program Lead & Medical Director, Genetic Medicines | Regeneron

Mohsin Ahmed, M.D., Ph.D., is Clinical Program Lead and Medical Director in Genetic Medicines at Regeneron. A board-certified psychiatrist and neuroscientist, his work focuses on the development of AAV gene therapies and siRNA therapeutics for central nervous system and auditory disorders. He leads programs from early development through registration, including the first FDA-approved gene therapy for hereditary hearing loss. Prior to joining Regeneron, he was an NIH-funded investigator and Assistant Professor of Psychiatry at Columbia University. Dr. Ahmed earned his M.D./Ph.D. in Neurobiology and Behavior from Columbia University and was awarded a Leon Levy Neuroscience Fellowship.
Brieana Buckley, PharmD, MS
Senior Vice President, Development and Program Leadership | Editas Medicine

Brieana Buckley, PharmD, MS, is Senior Vice President of Development and Program Leadership at Editas Medicine, where she leads program strategy, planning, and development activities across the it’s therapeutic pipeline. With over 20 years of experience in the healthcare and biotechnology industries, her expertise spans all phases of clinical development. She has contributed to ten successful commercial product launches and has extensive experience navigating global healthcare systems to improve access to innovative therapies designed to address unmet medical needs and transform patient outcomes. She holds a PharmD from the University of Utah and an MS in Pharmacoeconomics from the University of Florida.
Stephanie Cherqui, PhD
Professor of Pediatrics, Division of Genetics & Director of the UC San Diego Gene Therapy Initiative | UC San Diego

Stephanie Cherqui, PhD, 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 which 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.
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, PhD, 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 PhD 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.
Laurence Mignon, PhD
Vice President of Clinical Development | n-Lorem Foundation

Laurence Mignon, PhD, 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 PhD in Pharmacology and Experimental Therapeutics from Loyola University Chicago and conducted neurology research at University of California Los Angeles.
Alysson Muotri, PhD
Professor of Pediatrics, Cellular & Molecular Medicine & Co-Director of Gene Therapy Initiative | UC San Diego

Dr. Muotri is a professor at UC San Diego, Co-Director of the Gene Therapy Initiative, and Director at several university centers focused on stem cells, space, and evolutionary biology. His research focuses on brain evolution and modeling neurological diseases using human induced pluripotent stem cells and brain organoids. His awards include the prestigious NIH Director’s New Innovator Award & the NIH Replication Award, two Telly Awards for Excellence in Science Communication, among several others. Dr. Muotri earned a BSc in Biological Sciences and a Ph.D. in Genetics in Brazil. As a Pew Latin America Fellow at the Salk Institute, he pursued postdoctoral training in neuroscience and stem cell biology.
Leah Schust Myer
Founder & Executive Director | FamilieSCN2A Foundation

Leah Schust Myers is Founder & Executive Director of the SCN2A Foundation, where she leads efforts to advance research, patient support, and therapeutic development for individuals affected by SCN2A-related disorders. With over 20 years of experience in healthcare administration, her expertise spans healthcare operations, project management, communication, and community engagement. Following her son’s diagnosis with an SCN2A disorder, she became a leading advocate for the community. In her current role, she has helped accelerate research and raise awareness of SCN2A as a promising target for novel therapies through collaborations with clinicians, researchers, and industry partners.

Featured Panelists

Dan Oliver, MBA
CEO & Co-founder | 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 MBA from Harvard Business School and has degrees in Mechanical Engineering and Business from the California Institute of Technology.
Nicole Paulk, PhD
CEO & Founder | Siren Biotechnology

Nicole Paulk, PhD 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 BS in Microbiology, a PhD 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.
Karen Potter, PhD, JD
Partner | Morrison Foerster

Karen Potter, PhD, JD, is a nationally recognized patent attorney and leader of the Cell and Gene Therapy Patent Practice at Morrison Foerster in San Diego, California. With over 20 years of experience in life sciences intellectual property law, her practice focuses on patent strategy, prosecution, portfolio management, and strategic counseling for biotechnology and pharmaceutical companies worldwide. Her expertise includes cell and gene therapies, immunotherapies, vaccines, diagnostics, and protein therapeutics. Dr. Potter earned her PhD in cellular and molecular biology from Duke University and completed postdoctoral research at the La Jolla Institute for Allergy and Immunology.

Featured Chairs

Betty Cabrera, MPH
Director of Engagement and Operations, Gene Therapy Initiative | UC San Diego

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.
Donald Cleveland, PhD
Distinguished Professor of Cellular and Molecular Medicine | UC San Diego

Don Cleveland, Ph.D., is Professor of Cellular and Molecular Medicine at the University of California San Diego. A member of both the National Academies of Sciences and Medicine, his accomplishments include purifying and characterized tau, the first microtubule-associated protein linked to Alzheimer’s disease, uncovering mechanisms underlying major genetic forms of ALS, and developing gene-silencing therapeutics currently in clinical trials for ALS, Huntington’s, Parkinson’s, and Alzheimer’s diseases. He has received numerous honors, the most recent being the 2023 Breakthrough Prize in Life Sciences.
Masayuki Kai, PhD
Therapeutic Area Head (Hem/HemOnc), Research Strategy | Kyowa Kirin

Masayuki Kai, Ph.D., is Therapeutic Area Head for Hematology in the Global Research Organization at Kyowa Kirin, where he leads research strategy and portfolio development in hematology to advance innovative therapies. Dr. Kai has held a variety of research leadership positions across the Kyowa Kirin organization and earned his Ph.D. in Engineering from the Tokyo Institute of Technology in 2004 and was a Visiting Scientist at the Koch Institute for Integrative Cancer Research, MIT, from 2010 to 2011.
Noriko Kanaya, DVM, PhD
Principal Scientist | Nitto BioPharma

Noriko Kanaya, D.V.M., Ph.D., is a Principal Scientist at Nitto Biopharma, where she focuses on the development of cell-targeted lipid nanoparticles for gene editing applications. Her expertise in therapeutic development lies in translational pharmacology and the advancement of gene-editing therapeutics through in vivo preclinical research. She earned both her Doctor of Veterinary Medicine and Ph.D. degrees from the University of Tokyo.
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.