Randall Carpenter, MD
Chief Medical Officer
Dr. Carpenter’s career experience includes providing primary care to underserved populations, performing clinical and basic science research, and leadership positions in academic medicine, pharmaceutical and biotechnology companies. Most recently, he founded Seaside Therapeutics to leverage breakthrough scientific discoveries and develop therapeutics capable of correcting molecular perturbations that increase vulnerability to intellectual disability and autism. The experiences and knowledge gained establishing collaborations with expert clinicians, academic medical centers, scientists, patient advocacy groups, NIH, and biopharma are directly relevant to his role at RSRT and provide insights for effectively leveraging the expertise of a wide range of stakeholders.
He also authored and served as principal investigator on UO1 translational medicine grants jointly funded by NIMH, NINDS, NICHD, Autism Speaks and FRAXA, helped establish a fragile X clinical trial network, participated in NIH/FDA workgroups, and directly engaged FDA to define regulatory requirements for development of drugs to treat fragile X syndrome. He continues to be engaged in parallel and complementary efforts through participation in the advisory boards of EU-AIMS and the Translational Neuroscience Center of Boston Children’s Hospital and as a Research Affiliate in the Department of Brain and Cognitive Sciences at MIT.
While in industry, he led translational medicine teams responsible for 8 successful IND submissions and dozens of FDA-compliant clinical trials. Prior to joining industry, he held academic faculty appointments at Virginia Mason Medical Center, the University of Washington and Wake Forest University where he specialized in anesthesiology, pain management and translational medicine.
Progress toward a cure has been profound since I joined RSRT 4 years ago. AveXis/Novartis is currently advancing the gene replacement therapy toward clinical trials. In parallel, therapeutic programs to edit mutations in DNA and RNA or to reactivate the healthy but silent MECP2 gene are making significant progress. I am honored to represent the Rett community and look forward to advancing these therapeutic development programs as rapidly as possibleRandall Carpenter | July 2020