Dr. Berent is a veterinary internal medicine specialist/interventionalist who graduated from Cornell University and completed her residency at the University of Pennsylvania, where she served as an Adjunct Assistant Professor before joining the Animal Medical Center in NYC. She is the Director of the Interventional Endoscopy Service, focusing on clinical trials researching medical devices particularly for ureteral and biliary obstructions in animals with naturally occurring diseases. In 2014 Dr. Berent’s daughter, Quincy, was diagnosed with Angelman syndrome. In 2015 she joined the Board of Directors for the Foundation for Angelman Syndrome Therapeutics (FAST), becoming the Chief Science Officer. Dr. Berent serves as the co-director of the Angelman Syndrome Biomarker and Outcome Measure Consortium, to co-director for the International Angelman Syndrome Research Council (INSYNC-AS), and is an advisor to numerous pharmaceutical companies working on therapeutic candidates for rare neurodevelopmental disorders. Dr. Berent co-founded GeneTx Biotherapeutics, a company focused on advancing an antisense oligonucleotide therapy for AS, where she was the Chief Operating Officer. GeneTx was acquired in 2022 by Ultragenyx Pharmaceuticals, after launching the Phase1/2 clinical trial, and she now serves as a consultant for Ultragenyx. Dr. Berent is currently the Chief Development Officer at AS2 Bio accelerating numerous therapeutic programs for Angelman syndrome.
This session will examine the critical role of early-stage collaboration between advocacy groups and industry stakeholders in the development of orphan drugs and treatments for rare diseases. With the unique challenges posed by these conditions—including small patient populations, high development costs, and regulatory complexities—early engagement between patient advocates and pharmaceutical companies is essential to drive innovation, expedite regulatory pathways, and improve access to life-saving treatments.
Key topics will include the role of early dialogue in shaping clinical trials, the impact of advocacy efforts on regulatory approvals, and strategies for addressing the financial and logistical barriers that often impede progress in rare disease treatments. Participants will leave with practical strategies for fostering effective partnerships and lessons on how to leverage collaboration for meaningful impact in the orphan drug space.