Based on ‘hope and love,’ UB celebrates opening of FOXG1 Research Center

Hope and love aren’t often mentioned in the same breath as scientific research, but they’re exactly what UB’s FOXG1 Research Center (FRC) was founded on. 

“Love for every individual living with FOXG1 syndrome and other neurodevelopmental disorders, as well as their caregivers, and hope that we can find a cure for FOXG1 syndrome and related disorders on the basis of scientific discoveries we are making,” Soo-Kyung Lee, director of the FRC and the parent of a child with FOXG1 syndrome, told a crowd gathered to celebrate the center’s official opening Tuesday. 

The occasion included a ceremonial ribbon cutting and science symposium in the Buffalo Room of Capen Hall, as well as a tour of the Cooke Hall lab where the center’s researchers are developing a promising viral gene therapy against the rare disorder.

“This research center really represents a bold step forward, not only for the University at Buffalo but for the future of science,” said Robin Schulze, dean of the College of Arts and Sciences. “The center means we are that much closer to finding a cure for the neurodevelopmental disorder FOXG1 syndrome. Today’s ribbon cutting clears the way for innovative research and creative collaborations.”

The center is led by Lee, Empire Innovation Professor and Om P. Bahl Endowed Professor in the Department of Biological Sciences, College of Arts and Sciences, and her husband, Jae Lee, professor of biological sciences.

The Lees’ teenage daughter, Yuna, is one of only about 1,000 people in the world diagnosed with FOXG1 syndrome. It’s caused by a mutation of the FOXG1 gene, one of the most important genes for early brain development, and causes cognitive and physical disabilities, as well as life-threatening seizures. Those affected require 24/7 care.  

Since Yuna was diagnosed in 2012, the Lees, who joined UB in 2019, have dedicated their careers to studying the disorder. They’ve found that the FOXG1 gene and protein remain active in mice after birth, providing hope that some symptoms can be alleviated, and hope that therapy of the FOXG1 gene may be transferable to more common disorders like autism and Alzheimer’s disease.

This new center will support the Lees’ ongoing development of a viral gene therapy. A postnatal injection of the therapy into day-old mice rescued abnormalities in parts of the brain responsible for language, memory and social interaction. Their goal is to begin human clinical trials as early as spring 2026, pending U.S. Food and Drug Administration approval.

“[The Lees’] groundbreaking viral therapy offers renewed hope for patients and families affected by this rare disorder. Their personal journey and unwavering dedication to this cause are truly inspiring,” said Venu Govindaraju, vice president for research and economic development, whose office is supporting the center. 

Govindaraju added that between the FRC and the National AI Institute for Exceptional Education — which will use artificial intelligence to assist children with speech and language disorders — UB is dedicated to addressing the unique challenges faced by individuals with disabilities.

“The potential of artificial intelligence in drug discovery and personalized medicine is immense,” he said. “As a leading scientific research community and our excellence in AI and life sciences, we are committed to exploring innovative approaches that can accelerate the development of effective treatments for FOXG1 syndrome and other neurodevelopmental disorders.”

The FRC is also supported by the FOXG1 Research Foundation, where Soo-Kyung Lee is chief scientific officer. The foundation’s CEO, Nasha Fitter, who is also the parent of a child with FOXG1 syndrome, recalled first connecting with Soo-Kyung Lee via a Facebook group in 2017. 

“I honestly almost fell off my chair that we had a neuroscientist who was also a parent and no one had told me this,” Fitter said. “And then I learned that her husband was also a neuroscientist.”

Fitter and others co-founded the FOXG1 Research Foundation shortly after. She traced the organization’s rise, from its humble first symposium in 2018 to now having raised more than $7 million toward human clinical trials, and credited a large part of that success to its partnership with the Lees’ lab.

“They have built an entire gene therapy lab at the University at Buffalo. It is exceptional for an academic medical center to develop treatments,” Fitter said. “That does not usually happen at university campuses, and that is what is happening here.

“We’re literally creating the drug that will be given to our children in a clinical trial,” she added. “It’s a monumental step that this has happened in seven years.”

Jae Lee concluded the day by thanking children with FOXG1 syndrome for making their families whole.

“I feel we are closer to the finish line to find the cure for FOXG1 syndrome,” he said.