AS Research & Development Update, Part 1
Dan Harvey, chair of the Angelman Syndrome Foundation Academic Advisory Committee, introduces a conference focusing on the development of treatments for Angelman Syndrome. The conference features speakers from Ovid Therapeutics, ISIS Therapeutics, and Agilis Biotherapeutics. Ovid Therapeutics is developing a drug called Gaboxadol to address key symptoms of Angelman Syndrome. ISIS Therapeutics is working on antisense technology for the syndrome, aiming to find the best candidate drug for clinical trials. Agilis Biotherapeutics is focusing on DNA therapeutics for rare genetic diseases, including gene therapy for Angelman Syndrome, and is currently in the preclinical stage. The conference emphasises the importance of engagement and involvement in the treatment of Angelman Syndrome.
Dan Harvey welcomes attendees to the conference and introduces himself as the chair of the Angelman Syndrome Foundation Academic Advisory Committee. He also mentions that he works with DART Neuroscience, a company involved in the development of treatments for learning and memory disorders. Harvey welcomes the internet community, as the conference is being live streamed. He introduces his son Matthew, who has Angelman Syndrome, and shows pictures of him. Harvey explains that the focus of the conference will be on the development side of Angelman Syndrome research activities. He mentions the contributions of Harry Angelman, who first described Angelman Syndrome 50 years ago. Harvey discusses the progress made since the discovery of the UBE3A gene in 1997 and the development of treatments based on that understanding. He explains the three general approaches to the development of treatments for Angelman Syndrome: symptomatic treatments, studying the role of the UBE3A gene, and activating the paternal gene. Harvey introduces the panel of speakers: Matthew During from Ovid Therapeutics, Eugene Schneider from ISIS Therapeutics, and Jodi Cook from Agilis Biotherapeutics. Each panelist will give a brief overview of their organization’s work. Harvey emphasizes the importance of engagement and involvement in the treatment of Angelman Syndrome.
Matthew During discusses Ovid Therapeutics and their focus on developing a drug called Gaboxadol (Ovid 101) to address key symptoms of Angelman Syndrome. He explains the mechanism of action of Gaboxadol and how it restores tonic inhibition in the brain. During discusses the potential impact of Gaboxadol on seizures, movement and gait, sleep, and cognition. He presents preclinical data and studies that support the efficacy of Gaboxadol in improving these symptoms. During concludes by emphasizing the safety and tolerability of Gaboxadol and the potential benefits it can provide for individuals with Angelman Syndrome.
ISIS Pharmaceuticals is focused on antisense technology for Angelman Syndrome. They are working on finding the best candidate drug for clinical trials and collaborating with experts in the field.
Agilis Biotherapeutics is focused on DNA therapeutics for rare genetic diseases. They are working on gene therapy for Angelman Syndrome and are currently in the preclinical stage, conducting IND-enabling studies. Agilis is collaborating with Dr. Ed Weeber for gene therapy research. They are optimizing DNA components and vehicle for gene therapy and working on safety studies and clinical protocol development.
The speakers from Ovid Therapeutics, ISIS Therapeutics, and Agilis Biotherapeutics have provided an overview of their organizations’ work in developing treatments for Angelman Syndrome. The conference highlights the progress made in understanding the UBE3A gene and the potential for symptomatic treatments, gene therapy, and activating the paternal gene. Engagement and involvement in the treatment of Angelman Syndrome are emphasized as crucial for progress in the field.
- Title: AS Research & Development Update, Part 1
- Author(s): Dan Harvey, Matt During, Eugene Schneider, Jodi Cook
- Author(s)’ affiliation: Dart NeuroScience; Ovid Therapeutics; Ionis Pharmaceutical; Agilis Biotherapeutics
- Publication date: 2015-08-19
- Collection: 2015 ASF Family Conference