In 2014, the life of a successful veterinary specialist designing medical devices and enthralled with clinical research for animals came to a screeching halt. Her little girl, Quincy, 5.5 months old, was diagnosed with a rare neurogenetic condition called Angelman syndrome (AS). Through pure naiveté, scientific understanding and medical education, she knew a treatment for this condition was possible, despite there being no approved therapeutics. After countless conference calls, scientific stalking and a dream, Allyson Berent started her journey from becoming the chief science officer of the largest research funding foundation for AS in the world (Foundation for Angelman Syndrome Therapeutics [FAST]) to the cofounder and chief operating officer of GeneTx Biotherapeutics, a biotech company solely focused on developing an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome.
The foundation funded an academic laboratory under Scott Dindot at Texas A&M University to develop this ASO and deeply understand the unique phenomenon associated with AS (imprinting). This funding resulted in the discovery of a unique genetic region that could be exploited, essentially turning on a silent copy of the gene that is otherwise missing in the neurons of those with AS. It was at that time that the foundation decided to license this technology and develop this drug, recruit some of the most experienced experts in ASO drug development in the world and drive the timeline for a first in human clinical trials.
Through personal determination, supportive expertise and amazing consultants, GeneTx Biotherapeutics completed the US Food and Drug Administration’s Investigational New Drug (IND) program enabling studies from the first exploratory in vivo studies to the last in vivo good laboratory practice (GLP) toxicity studies in exactly 13 months. The vision and dedication of parents and a strong community drove this program, allowing the team to remain singularly focused on a population of approximately 1:15,000.
Allyson Berent