From parent advocate to nonprofit chief science officer, to biotherapeutic company cofounder — A personal journey through drug development for Angelman syndrome

Speaker Allyson Berent, DVM, DACVIM, COO and Cofounder, GeneTx Biotherapeutics
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Webinar

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On May 29, 2020, Allyson Berent discussed the journey that led her to becoming chief science officer of the largest research funding foundation for Angelman syndrome in the world (Foundation for Angelman Syndrome Therapeutics [FAST]), and the cofounder and chief operating officer of GeneTx Biotherapeutics — a biotech company solely focused on developing an antisense oligonucleotide for the treatment of Angelman syndrome — after her daughter was diagnosed with Angelman syndrome at 5.5 months old.

About the Webinar

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.

About the Speaker

Allyson Berent graduated from Cornell University College of Veterinary Medicine in 2002. She completed an internship at the University of Minnesota and a residency in internal medicine at the Veterinary Hospital of the University of Pennsylvania thereafter. She was boarded in internal medicine in 2006.

After completing a fellowship in interventional radiology at the Veterinary Hospital of the University of Pennsylvania and a fellowship in endourology at Thomas Jefferson University, she served as adjunct assistant professor in internal medicine and interventional radiology/interventional endoscopy at the Matthew J. Ryan Veterinary Hospital of the University of Pennsylvania. Since 2009, she has held a staff position at the Animal Medical Center in Interventional Radiology and Endoscopy and has been director of the Interventional Endoscopy Services. Her research interests are in minimally invasive diagnostics and therapeutics and medical device development including endourology, ureteral disease, gastrointestinal interventions, nasopharyngeal stenting and stem cell treatment for kidney disease. Her primary research is in medical device development.

In 2015, Berent became the chief science officer for the Foundation for Angelman Syndrome Therapeutics and has been integral in driving the research portfolio in novel treatment options for this rare genetic disorder. In 2017, Berent became the chief operating officer of GeneTx Biotherapeutics, a company with a sole mission to advance antisense oligonucleotide therapies toward human clinical trials for the treatment of Angelman syndrome.

Past Lectures

What do we mean by ‘autism risk genes’?

David Ledbetter, Ph.D.
Chief Clinical Officer, Dascena

Joseph Buxbaum, Ph.D.
Director, Seaver Autism Center
Professor, Psychiatry, Neuroscience, Genetics and Genomic Sciences
Vice Chair for Research and Vice Chair for Mentoring, Psychiatry, Icahn School of Medicine at Mount Sinai

Heather Mefford, M.D., Ph.D.
Full Member, St. Jude Children’s Research Hospital

David Ledbetter and Joseph Buxbaum discussed whether there are genes for which mutations confer risk specific to autism or whether these genes are really conferring general risk of disrupted brain development. The discussion was moderated by Heather Mefford.

Small molecules, genes and antisense oligonucleotides: Industry perspectives on treatment development for ASD

Federico Bolognani, M.D., Ph.D.
Vice President, Head of Clinical Science, Axial Therapeutics

Stuart Cobb, Ph.D.
Chief Scientific Officer, Neurogene; Research Fellow, University of Edinburgh

Yael Weiss, M.D., Ph.D.
Vice President, Business Development, Ultragenyx

Randy Carpenter, M.D.
Chief Medical Officer, Rett Syndrome Research Trust; Co-Founder, Allos Pharma

Federico Bolognani, Stuart Cobb, and Yael Weiss joined a panel to discuss new industry developments on the use of small molecules, gene therapy and antisense oligonucleotides as treatment approaches for autism spectrum disorders (ASD). The panel discussion was moderated by Randall Carpenter.

New research results from the Australian Autism Biobank study

Jake Gratten, Ph.D.Group Leader, Mater Research Institute, The University of Queensland
Adjunct Senior Research Fellow, Institute for Molecular Bioscience
Naomi Wray, Ph.D.National Health and Medical Research Council Leadership Fellow – Group Leader, Institute for Molecular Bioscience
Affiliate Professor, Queensland Brain Institute, The University of Queensland

Jake Gratten and Naomi Wray presented findings from the Australian Autism Biobank study, an initiative to establish an Australian resource of biospecimens, phenotypes and genomic data for autism research.

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