News Briefs

Dr. Jonathan Mink receives Child Neurology Society’s highest honor

Jonathan W. Mink, M.D., Ph.D., the Frederick A. Horner M.D. Distinguished Professor in Pediatric Neurology, and Chief of Child Neurology at University of Rochester Medical Center, has been awarded the Child Neurology Society’s (CNS) 2021 Hower Award.

The award is the organization’s highest honor and is given annually to a child neurologist recognized as an outstanding teacher and scholar, and for making high levels of contributions to the field and the CNS. Mink served as president of CNS from 2017 to 2019.

In his early career, Mink studied the neurobiological basis of Movement Disorders and developed the prevailing model of how the basal ganglia of the brain contribute to motor control. Since moving to the University of Rochester in 2001, he has focused on clinical research on Movement Disorders, including Tourette syndrome and Batten disease. Mink is a dedicated teacher and mentor and many of his students have gone on to become child neurologists and physician-scientists. He is currently the director of the University of Rochester Batten Center, co-director of the University of Rochester Tourette Center of Excellence, and co-director of the University of Rochester Intellectual and Developmental Diseases Research Center.

He was recognized at the society’s annual meeting, where he delivered the Hower Award lecture entitled, “On Mentors and Protégés: Standing on the Shoulders of Giants and Following Footsteps into the Future.”

“Receiving the Hower Award is a tremendous honor,” Mink said. “I have had the privilege to learn from so many giants and to learn even more from the students, residents, and fellows I have mentored. Each protégé has led me to explore new questions and new directions. The contributions recognized by this award would have not been possible without their inspiration and partnerships.”

The Child Neurology Society is the preeminent non-profit professional association of pediatric neurologists in the United States, Canada, and worldwide.

URMC to lead first gene therapy study for CLN5 Batten disease

The University of Rochester Medical Center (URMC) will serve as the lead study site in the U.S. for an investigational treatment being developed by Neurogene Inc. for CLN5 Batten disease, a rare and fatal neurodegenerative disorder.

“This trial will move research forward in developing a potentially disease-modifying treatment for CLN5 disease, providing hope to individuals and families where currently none exists,” said Jonathan W. Mink, M.D., Ph.D., the Frederick A. Horner M.D. Distinguished Professor in Pediatric Neurology, and Chief of Child Neurology at URMC. The Medical Center is home to the University of Rochester Batten Center (URBC), which is led by Mink and is one of the nation’s premier centers dedicated to the study and treatment of Batten disease.

The planned open-label phase 1/2 clinical trial will evaluate a one-time treatment via intracerebroventricular (ICV) and intravitreal (IVT) routes of administration. The treatment, called NGN-101, uses an adeno-associated virus to deliver a healthy version of the CLN5 gene directly to the central nervous system and the eye. Animal studies have demonstrated that the treatment has the potential to halt the key features of disease progression, including vision, motor, cognitive, and behavioral declines.

Neurogene’s NGN-101 was recently cleared by the Food and Drug Administration to begin clinical trials and has received Orphan Drug Designation by the U.S. and European regulatory agencies. URMC anticipates enrolling the first study participants in the first half of 2022.

For more information: rochesterbatten.urmc.edu