The Use of Gene Therapy for the Production of UBE3A in the Treatment of Angelman Syndrome: Discussion of Future Clinical Trials
Agilis Biotherapeutics is working on gene therapy for the treatment of Angelman Syndrome. They are focused on developing a viable therapeutic using DNA therapeutics and gene therapy. They are using adeno-associated virus vectors to deliver the therapeutic to the brain. Gene therapy has seen significant advancements in recent years, with clinical successes and improved manufacturing capabilities. Agilis aims to have a lead candidate for gene therapy for Angelman Syndrome by 2016 and plans to move into clinical development. They are also working on establishing reliable outcome measures for the evaluation and registration of therapeutics for Angelman Syndrome.
The use of gene therapy for the treatment of Angelman Syndrome has been a long-awaited development in the field. Agilis Biotherapeutics is one of the companies at the forefront of this research, and they presented their work at the 2015 FAST Science Summit. In this talk, we will discuss the highlights of their presentation and their plans for future clinical trials.
Agilis Biotherapeutics: A Dedicated Approach
Agilis Biotherapeutics is a company dedicated to advancing DNA therapeutics for rare genetic disorders of the central nervous system (CNS). They specialize in gene therapy and are focused on developing a viable therapeutic for Angelman Syndrome. The company has a team of experienced scientists and advisors who are working towards optimizing the DNA construct and delivery platform for their therapeutic.
The Promise of Gene Therapy
Gene therapy has seen significant advancements in recent years, with clinical successes in various genetic diseases. The regulatory environment has also become more favorable, with the Orphan Drug Act providing benefits for gene therapy development. Additionally, manufacturing challenges have been addressed, making gene therapy a more viable option. The value creation in the gene therapy space has attracted investment, further driving progress in the field.
Understanding Angelman Syndrome
Angelman Syndrome is a genetic disorder caused by the loss or mutation of the UBE3A gene, resulting in a decrease in the expression of the E6AP protein. This leads to impaired cognitive function and other symptoms associated with the syndrome. Agilis Biotherapeutics has licensed the worldwide exclusive rights to the Angelman syndrome gene therapy and is working on optimizing the human UBE3A gene construct for therapeutic development.
Gene Therapy for Angelman Syndrome
Agilis Biotherapeutics is using adeno-associated virus (AAV) vectors for gene delivery. These vectors are safe and well-tolerated, and they can target specific cells in the central nervous system. The goal is to deliver the UBE3A gene construct to the cells that need it, allowing for the expression of the E6AP protein and restoring function. Preclinical studies using mouse models have shown promising results, and the company is actively working on optimizing the DNA and delivery methods.
The Drug Development Process
Agilis Biotherapeutics is currently in the preclinical stage of drug development. They are conducting safety studies and process development to ensure the therapeutic is safe and can be manufactured consistently. Once they have a lead candidate, they will engage in regulatory discussions and plan for Phase 1 clinical trials. The company is committed to working with the Angelman Syndrome community to develop outcome measures and biomarkers that can be used for therapeutic evaluation and registration.
The Outcome Consortium Mission
Agilis Biotherapeutics recognizes the importance of establishing reliable outcome measures and biomarkers for Angelman Syndrome. They are planning to convene a planning meeting with stakeholders to discuss short-term and long-term outcome measures. The goal is to develop measures that can be used for therapeutic development, evaluation, and registration. The company is also open to collaboration and funding opportunities to support this mission.
Agilis Biotherapeutics is dedicated to advancing gene therapy for the treatment of Angelman Syndrome. They are working on optimizing the DNA construct and delivery platform and plan to move into clinical development in the near future. The company is committed to working with the Angelman Syndrome community and other stakeholders to develop outcome measures and biomarkers that can facilitate therapeutic evaluation and registration.
- Title: The use of gene therapy for the production of UBE3A in the treatment of Angelman Syndrome. Discussion of future clinical trials
- Author(s): Mark Pykett, Jodi Cook, Gregory Robinson
- Author(s)’ affiliation: Agilis Biotherapeutics
- Publication date: 2015-12-04
- Collection: 2015 FAST Science Summit