Edwin Weeber, PhD
Head of Research and Discovery*
Ed has a long history not just with Angelman syndrome, but with FAST. To start with, he led pioneering initiatives aimed at ameliorating the symptoms in the Angelman syndrome mouse model. Through a multifaceted approach encompassing genetic interventions, protein supplementation strategies, and gene therapy techniques, his groundbreaking work shed light on the underlying mechanisms of the disorder and the potential for therapeutic intervention. In addition, he helmed the first AS consortium of scientists from different specialties and academic laboratories to collaborate and focus on discovering potential therapies for AS. This collaborative effort was dedicated to the development of promising therapeutic avenues.
A message from Ed: My experience with Angelman syndrome has bridged over 20 years, starting as a postdoctoral researcher in the Division of Neuroscience at Baylor College of Medicine and being one of the first researchers to report on the molecular changes underlying UBE3A deficiency in 2003. While at Vanderbilt University Medical Center, we reported the first genetic rescue of the Angelman syndrome mouse model in 2007. After moving my laboratory to the University of South Florida, we were the first to report the use of an Adeno-Associated Virus (AAV) to replace the missing Ube3a gene in the AS mouse model in 2011. I was honored to lead the FAST F.I.R.E. initiative in 2012.
In 2019, I accepted a position at a pharmaceutical company to facilitate the development of an investigational AS gene replacement therapy. My involvement in understanding the molecular mechanisms and potential therapeutic strategies to treat Angelman syndrome has allowed me to work with some of the best and brightest researchers in the field and meet with hundreds of families in the AS community worldwide. To say working for the families and individuals living with Angelman syndrome is an honor would be a true understatement. Returning to FAST in the capacity of Head of Research and Discovery feels like coming home. Witnessing the profound impact FAST has had on advancing drug development for Angelman syndrome over the past few years is awe-inspiring. Now, I'm determined to channel all my efforts into maximizing this impact, ensuring that we continue to make strides towards improving the lives of everyone affected by Angelman syndrome.
How will you support FAST’s mission? As the Head of Research and Discovery, I will be working directly with the Chief Science Officer, Dr. Allyson Berent, to align FAST’s discovery efforts with the mission of the organization. My role will ensure that continued scientific rigor, innovation, and novel opportunities are leveraged, with a focus on promising potential therapeutic strategies that can hopefully be translated to patients. I will be working directly with the Director of Science, Dr. Nycole Copping, and the Chair of the Scientific Advisory Board, Dr. Barbara Bailus, to review ongoing and future research projects, identify scientific breakthroughs that can potentially be focused on AS, and synthesize the complexity of all of the robust ongoing AS research.
What did you do before FAST? I left academia in 2019 to work at PTC Therapeutics, a mid-sized pharmaceutical company where I was able to contribute to their work on numerous rare neurological disorders at different stages of development. I learned firsthand how a therapeutic can be moved efficiently through the pipeline of early discovery to first-in human without sacrificing high-quality science. When these initiatives were unfortunately discontinued in 2023, I also learned how fragile the process can be, and how important a role there is for FAST to play.
5 fun facts about you:
I met my wife Jessica, who also has a PhD in Neuroscience, in Houston while I was a postdoctoral researcher. We have a son Andrew, 18, and a daughter Keira, 16.
We also have a plethora of pets consisting of two dogs, a minipig, an axolotl, a spotted gecko, two crested ducks, and a pygmy goat. (No, we do not live on a farm.)
I’m a shade tree mechanic. My passion for automobiles was put in the “backseat” with the addition of kids. Now that they are older, I enjoy working on my cars - with much of my free time eaten up by my Mustang GT project.
I enjoy movies of all genres and routinely watch horror movies with my daughter. However, I have never seen Dances with Wolves!
I have lived in eight cities in six different states.