CAMBRIDGE, MA: Agilis Biotherapeutics, a biotechnology company announces a license agreement with the University of South Florida (USF) for the treatment of Angelman Syndrome (AS). Under the terms of the agreement, Agilis will be granted worldwide, exclusive rights to AS gene therapy candidates and related intellectual property.

The treatment process will leverage gene therapy technology developed by Edwin Weeber, PhD, Director of the Neurobiology of Learning and Memory and Chief Scientific Officer at the USF Health Byrd Alzheimer's Research Institute at the University of South Florida.

The alliance will focus the genetic malfunctions of AS using gene therapy to deliver a corrective UBE3a gene to rescue neurological deficits in patients suffering from this rare disease. Dr. Weeber explains, “Our research has demonstrated that restoration of UBE3a function has the potential to address many of the neurological symptoms of AS, and to positively impact the quality of life by addressing Angelman’s CNS manifestations.”

 “We are delighted to be working with Dr. Weeber in this exciting area of gene therapy,” says Greg Robinson, PhD, Agilis’ Chief Scientific Officer. “Dr. Weeber’s science is compelling and provides an excellent platform from which to develop an effective UBE3a therapy for patients with AS, for whom no effective treatment options currently exist.”

AS, a rare genetic disorder affecting the central nervous system and its features include delayed development, intellectual disability, severe speech impairment, seizures and ataxia.

On the partnership, Dr. Mark Pykett, President and Chief Executive Officer of Agilis, comments, “Adding this program to our pipeline enhances our corporate mission of developing functional cures for rare genetic diseases of the CNS. As we expand our programs, we are continually augmenting our differentiated technologies, skills and capabilities in addressing rare CNS disorders with innovative DNA therapeutic strategies.”