Accelerating preclinical development for AAV based gene therapies: bridging the gap between discovery and clinical development

Cell & Gene Therapy Insights 2021; 7(1), 127–130


Published: 24 February 2021
Louise Rodino-Klapac

Dr. Rodino-Klapac is a gene therapy pioneer who has dedicated her professional life to advancing medicines designed to treat genetically based diseases. With professional experience across industry and academia, she is renowned for her contributions to molecular genetics and gene therapy that have advanced the field. She is author to a vast body of published, peer-reviewed work, the recipient of multiple awards, a National Institutes of Health (NIH) Fellow appointee, and current Board member of the Association for Regenerative Medicine, as well as a member of the American Society for Gene and Cell Therapy, the American Academy of Neurology, and the American Association for the Advancement of Science. She is the former head of the Laboratory for Gene Therapy Research at Nationwide Children’s Hospital, established the Gene Therapy Center of Excellence within Sarepta and leads the Company’s Gene Editing Innovation Center, directing a team of researchers to discover and develop novel gene replacement and gene editing therapies. She co-founded and served as chief scientific officer of Myonexus Therapeutics, a gene therapy company focused on limb-girdle muscular dystrophies acquired by Sarepta in 2019. She currently serves as Sarepta’s Executive Vice President, Chief Scientific Officer. Her work has led to 11 investigational new drug applications and she is the co-inventor of SRP-9001, an investigational micro-dystrophin gene therapy, and the inventor of five investigational gene therapies for limb-girdle muscular dystrophy. She is the inventor of over 50 U.S. and over 70 international published patent applications. She earned her Ph.D. in molecular genetics from the Ohio State University and graduated summa cum laude from Kings College in Wilkes-Barre, Pennsylvania, with a Bachelor of Science degree in Biology.