GeneTx Biotherapeutics: A Novel Approach to Drug Development – The Why, The What, and The How?

Quick Overview

GeneTx Biotherapeutics is a biotechnology company focused on developing a novel therapeutic approach for Angelman Syndrome. The company was founded by Paula Evans, the CEO of GeneTx and the chairperson of the Foundation for Angelman Syndrome Therapeutics (FAST). The company aims to develop an antisense oligonucleotide (ASO) drug that can reactivate the paternal allele of the UBE3A gene, which is responsible for Angelman Syndrome. The ASO works by targeting and cutting the UBE3A antisense transcript, allowing the paternal allele to be turned back on and produce the UBE3A protein. GeneTx has conducted in vitro and in vivo studies to evaluate the efficacy and safety of the ASO. The company is working towards submitting an Investigational New Drug (IND) application to the FDA to start clinical trials. GeneTx is also seeking partnerships with established pharmaceutical companies to further develop and commercialize the ASO drug.


In this talk, we will discuss the groundbreaking work being done by GeneTx Biotherapeutics in the field of drug development for Angelman Syndrome. We will introduce the key individuals involved in this research and provide an overview of their approach and progress so far.

The Team

The team at GeneTx Biotherapeutics is led by Paula Evans, the founder of the Foundation for Angelman Syndrome Therapeutics (FAST) and the CEO of GeneTx Biotherapeutics. Scott Dindot, an associate professor at Texas A&M University, is the scientific consultant and inventor for the project. Jennifer Panagoulias, a regulatory consultant with over 20 years of experience in drug development, provides expertise in advancing global development programs for rare neurological diseases.

The Why

The motivation behind starting GeneTx Biotherapeutics was to find a way to develop a treatment for Angelman Syndrome. The team recognized the potential of an antisense therapeutic approach to target the paternal allele and circumvent the antisense transcript that shuts it down. They realized that pharmaceutical companies were not interested in early-stage proof of concept research, so they decided to start their own biotechnology company to attract investor funds and develop this novel approach.

The What

GeneTx Biotherapeutics is focused on developing an antisense oligonucleotide (ASO) drug to treat Angelman Syndrome. ASOs are short, synthetic polymers that target specific RNA sequences and alter their expression. By designing ASOs to target the antisense transcript that turns off the paternal allele, GeneTx aims to reactivate the silenced gene and restore UBE3A protein production in neurons.

The How

To develop their ASO drug, GeneTx Biotherapeutics conducted extensive in vitro and in vivo studies. They identified a specific target within the antisense transcript and designed ASOs to bind to and cut the RNA, thereby stopping its transcription. In in vitro studies using human neurons, they observed a significant knockdown of the antisense transcript and an increase in UBE3A levels.

The IND Application

GeneTx Biotherapeutics is currently working towards submitting an Investigational New Drug (IND) application to the FDA. The IND application contains detailed information about the drug, the clinical plan, the manufacturing process, and the pharmacology and toxicology data. It is crucial to establish a good relationship with the FDA and address their concerns during the pre-IND meeting to ensure a smooth review process.


GeneTx Biotherapeutics is dedicated to developing successful treatments for individuals with Angelman Syndrome. They are committed to collaborating with other industry partners and sharing data and learnings to advance the field. Their ultimate goal is to partner with an established company that can help bring their ASO drug to human clinical trials and achieve their mission of improving the lives of those affected by Angelman Syndrome.

Talk details

  • Title: GeneTx Biotherapeutics. A Novel Approach to Drug Development – The Why, The What, and The How?
  • Author(s): Paula Evans, Allyson Berent, Scott Dindot, Jennifer Panagoulias
  • Author(s)’ affiliation: FAST; GeneTx Biotherapeutics
  • Publication date: 2019-01-10
  • Collection: 2018 FAST Science Summit