Spotlight Symposium honors Lee’s personal, purposeful FOXG1 research

Many parents feel helpless when their child is diagnosed with a debilitating neurodevelopmental disorder.

For neurodevelopmental biologist Soo-Kyung Lee, her child’s diagnosis felt like a call to action.

“I suddenly realized the research I had been doing my entire career …  might be the only thing that could someday help my daughter,” Lee told a crowd of researchers in Lippes Concert Hall.

Lee was honored at the second Spotlight Symposium last week for her work directing the FOXG1 Research Center at UB and developing a drug to treat FOXG1 syndrome, now in clinical trials.

The Spotlight Symposia Series recognizes and elevates College of Arts and Sciences faculty by inviting distinguished speakers to campus who share their research interests. This year’s symposium, “Master Switches of the Mind: Gene Regulation and Neurodevelopmental Disorders,” honored Lee by bringing together researchers who study how genes like FOXG1 affect brain development.

Jeff Grabill, dean of the college, called Lee a “trailblazer” whose work highlights the power of discovery-based research to advance innovation and change lives.

“Her work is much more than a curiosity or normal research endeavor. Her work is personal,” he said. “It is purposeful and it is full of heart.”

Derek Daniels, professor and chair of the Department of Biological Sciences, added that Lee’s research not only demands scientific understanding of brain development, but also demonstrates how basic science can drive translational progress. 

“This work exemplifies what makes biological sciences at UB so impactful: deep expertise, interdisciplinary collaboration and a commitment to research that improves lives,” he said.

Lee, SUNY Empire Innovation Professor and the Om P. Bahl Endowed Professor of Biological Sciences, recounted growing up in South Korea, falling in love with genetics and eventually landing her first faculty position at Baylor College of Medicine in Houston. 

“I thought I knew how my life was going to go,” she said. “But I was wrong. Life doesn’t go as planned.”

Lee’s daughter, Yuna, was diagnosed with FOXG1 syndrome in 2012 at the age of 2. It’s caused by mutations in the Forkhead Box G1, one of the most important genes for early brain development. It’s also rare, with just 1,500 known patients worldwide.  

Lee said the news wasn’t devastating — it was clarity. For almost two years, she and her husband, Jae Lee, professor of biological sciences, had been searching for answers about why baby Yuna wasn’t developing as expected. 

“For the first time, we had a name and a place to aim,” Lee said. 

The Lees shifted their research focus to the basic science behind FOXG1 syndrome and the search for treatment. They joined UB in 2019 and have since developed a viral gene therapy designed to address the underlying genetic cause of the disorder. 

Their therapy, FRF-001, delivers a functional copy of the FOXG1 gene using an adeno-associated virus 9 (AAV9) vector. It has been shown to reverse some abnormalities in mice with FOXG1 syndrome, including in parts of the brain associated with language, memory and social interaction. 

A child with FOXG1 syndrome received the therapy for the first time earlier this month as part of clinical trials cleared by the U.S. Food and Drug Administration. 

“It’s a beginning, not an ending,” Lee said. “The beginning is a beautiful thing when you’ve been waiting for 14 years.”

The Lees’ FOXG1 Research Center is supported by the Office of the Senior Vice President for Research, Innovation and Economic Development. 

“The work being done at the FOXG1 Research Center represents the very best of what we strive for at UB,” said Senior Vice President Venu Govindaraju. “It reminds us that research isn't just about data points or publications — it's about the actual impact we have on human lives.” 

The research center, as well as the clinical trial, are also supported by the FOXG1 Research Foundation. It’s believed to be the first instance of a parent-led, rare disease nonprofit foundation independently sponsoring its own multisite, international gene therapy clinical trial.

“We have been able to do it because of you,” FOXG1 Research Foundation co-CEO Nasha Fitter told Lee in a video message. “For that, I and the entire FOXG1 community are forever grateful and forever in your debt.”

The keynote address was delivered by Lee’s “science hero” and former colleague at Baylor College of Medicine, Hugo Bellen. One of the world’s premier researchers in fruit fly genetics, Bellen explained how fruit flies are used as a model organism to study human disease because many of their genes have human counterparts. 

Bellen’s talk underscored how rare pediatric neurological diseases like FOXG1 syndrome can drive discoveries in more common diseases. The FOXG1 gene has been linked to autism spectrum disorder, Alzheimer’s disease and certain cancers, raising the possibility that Lee’s therapy could have broader applications. 

Lee concluded her address by thanking UB for supporting her research.

“Universities make choices about what they care about. UB has made a very important statement about what kind of institute it wants to be,” she said.

She also thanked her family for their support, including Jae and their 13-year-old son, Joon. She credited Yuna for teaching her that “science can be a form of love.”

“I didn’t know so much love can be delivered through science,” she said.