UF Health researcher’s gene therapy technique being further developed to treat neurodegenerative disorders

A gene therapy technique discovered by a University of Florida Health researcher and her colleague is now being used to develop treatments for neurodegenerative disorders.

During gene therapy, engineered viruses are used to deliver new genes to a patient’s cells. But in some patients, the immune system recognizes and fights the helpful adeno-associated virus, or AAV. A redesigned virus that can slip past the immune system was successfully tested in human and animal serum in 2017.

The technique was developed by Mavis Agbandje-McKenna, Ph.D., a professor in the UF College of Medicine’s department of biochemistry and molecular biology and director of the Center for Structural Biology. Her collaborator is Aravind Asokan, Ph.D., who was an associate professor of genetics at the University of North Carolina when the research was conducted.

Now, the antibody-evading viruses are part of a collaboration involving two companies that will further develop therapies for Friedrich’s ataxia and two additional undisclosed neurological disorders. Friedrich’s ataxia is a progressive movement disorder caused by a genetic mutation. It leads to a loss of sensation in the limbs and problems with speech and walking.

The collaboration of the two companies, StrideBio Inc. and Takeda Pharmaceutical Co., will attempt to further develop the novel virus protein shells, making them more potent and able to evade neutralizing antibodies. The intellectual property used in this collaboration is jointly owned by UF and the University of North Carolina, and licensed to StrideBio.