Agilis Biotherapeutics: Advancing Gene Therapy for Angelman Syndrome

Quick Overview

Agilis Biotherapeutics is a biotech company working on gene therapy for Angelman syndrome. They have partnered with Dr. Ed Weeber at the University of South Florida to develop a gene therapy program for the disorder. Agilis has filed for orphan product designation in the US and EU and has evaluated different routes of administration for the therapy. They are currently working on optimizing the therapy and conducting safety studies in larger animals. Agilis is also collaborating with other organizations in the field to develop biomarkers and outcome measures for clinical trials. They are committed to finding a treatment for Angelman syndrome and encourage families to join the Worldwide Registry and stay informed about the progress of gene therapy.

Introduction

Agilis Biotherapeutics is a biotech company dedicated to advancing innovative gene therapies for rare genetic diseases of the central nervous system. They have partnered with Dr. Ed Weeber at the University of South Florida to develop a gene therapy program for Angelman syndrome. In this talk, we will discuss the progress made by Agilis and the FIRE team in pushing gene therapy forward for Angelman syndrome.

The Journey So Far

Agilis began their work in Angelman syndrome in March 2015. They have been working closely with Dr. Ed Weeber, a professor and director of the Neurobiology and Learning and Memory Laboratory at the University of South Florida. Together, they have made significant progress in developing a gene therapy for Angelman syndrome.

Understanding Agilis Biotherapeutics

Agilis Biotherapeutics is focused on developing gene therapies for rare genetic diseases of the central nervous system. They have a pipeline of diseases they are actively working on, including AADC deficiency, Friedreich’s ataxia, Angelman syndrome, and cognitive disorders. Agilis is committed to staying focused on these disease indications to take learnings from one disease and apply them to another.

The Promise of Gene Therapy

Gene therapy is a novel way to deliver a corrected piece of DNA into a patient. Agilis uses adeno-associated virus (AAV) gene therapy, which is a safe and effective method. The AAV virus is used as a vehicle to deliver the corrected gene to the targeted cells in the brain. Agilis has seen promising results in animal studies, and they are now working on optimizing the delivery and dosage of the therapy.

The Success of AADC Gene Therapy

Agilis has had success with gene therapy for AADC deficiency, a disease that affects dopamine production. They have treated children with AADC deficiency and have seen significant improvements in their motor and cognitive abilities. The therapy has been durable for up to five years, and the children are now able to take advantage of educational services and therapies.

Moving Forward with Angelman Syndrome Gene Therapy

Agilis is now focused on developing gene therapy for Angelman syndrome. They have successfully tested the therapy in mouse models, and they are now working on optimizing the route of administration and conducting safety studies in larger animals. They are also collaborating with the Angelman community to develop outcome measures and biomarkers for future clinical trials.

What Can You Do?

As a parent or caregiver, you can participate in the Worldwide Registry for Angelman Syndrome, which is a valuable resource for researchers and clinicians. You can also stay informed about the latest developments in gene therapy for Angelman syndrome by keeping in touch with advocacy groups and participating in clinical trials when they become available.

Conclusion

Agilis Biotherapeutics is dedicated to advancing gene therapy for Angelman syndrome. They have made significant progress in developing a therapy and are now working on optimizing the delivery and dosage. With the support of the Angelman community and ongoing research, they are hopeful that gene therapy will provide a promising treatment option for individuals with Angelman syndrome.