Dr. Allyson Berent, DVM, DACVIM, is a veterinary internal medicine specialist who serves as the Director of Interventional Endoscopy Services at the largest animal hospital in the world, The Animal Medical Center, in New York City. After graduating from Cornell University College of Veterinary Medicine she completed an internship at the University of Minnesota and a residency in Small Animal Internal Medicine at the Veterinary Hospital of the University of Pennsylvania. After completing a fellowship in interventional radiology at the Veterinary Hospital of the University of Pennsylvania, a fellowship in Endourology at Thomas Jefferson University, and an Interventional radiology fellowship at the Hospital of the University of Pennsylvania, she served as an Adjunct Assistant Professor in Internal Medicine and Interventional Radiology/ Interventional Endoscopy at the Matthew J. Ryan Veterinary Hospital of the University of Pennsylvania. Dr. Berent has a particular focus on medical device development, stem cell therapy through regenerative medicine and selective arterial delivery, ureteral diseases, urinary incontinence and minimally invasive management of upper tract urinary obstructions and biliary obstructions. In 2014 Dr. Berent’s daughter was diagnosed with a rare non-degenerative neurogenetic disorder called Angelman syndrome. In October of 2015 she joined to Board of Directors as a Scientific Director for the Foundation for Angelman Syndrome Therapeutics (FAST), and in March of 2016 became the Chief Science Officer for the Foundation. Dr. Berent helped to spearhead the development of a pre-competitive biomarker and outcome measure consortium in order to bring patient focused outcome measures forward for human clinical trials (Angelman Syndrome Biomarker and Outcome Measure Consortium-ABOM) and Co-Founded the International Angelman Syndrome Research Council (INSYNC). Through FAST, Dr. Berent collaborated with a consortium of scientists to encourage translational research opportunities, in order to help bring novel genetic therapies forward toward human clinical trials. Through this work, with the foundation, Dr. Berent co-founded GeneTx Biotherapeutics, a company singularly focused to advance an antisense oligonucleotide (ASO) therapy through IND enabling studies and a phase 1/2 clinical trial. Dr. Berent currently serves as the Chief Operating Officer of GeneTx Biotherapeutics, who partnered with Ultragenyx Pharmaceuticals in August of 2019. The Phase 1/2 clinical trial started enrolling patients in February 2020 as the first intrathecally delivered ASO for Angelman syndrome, a study of safety and tolerability of GTX-102.
Elizabeth Berry-Kravis MD, PhD is a Professor of Pediatrics, Neurological Sciences, and Biochemistry at Rush University Medical Center in Chicago. She established the Fragile X Clinic and Research Program in 1991, through which she provides care to over 700 patients with fragile X syndrome (FXS). She has studied medical issues, epilepsy and psychopharmacology in FXS, and has been a leader in translational research in FXS including development of outcome measures and biomarkers, natural history studies, newborn screening, and particularly clinical trials of new targeted treatments in FXS, and her laboratory studies the cellular role of fragile X mental retardation protein (FMRP), relationship between FMRP and clinical function, and optimization of genetic testing methods. More recently she has expanded clinical and translational work to other neurodevelopmental disorders and genetic neurodegenerative diseases including autism spectrum disorders, Phelan McDermid syndrome, Rett syndrome, Angelman syndrome, Niemann-Pick type C, Battens disease, pantothenate kinase-associated neurodegeneration, and creatine transporter deficiency. She has received the NFXF Jarrett Cole Clinical Award, FRAXA Champion Award, NFXF William and Enid Rosen Research Award, March of Dimes Jonas Salk Research Award, American Academy of Neurology Sidney Carter Award in Child Neurology and John Merck Fund Sparkplug Award.