Rare Disease Day at University at Buffalo

Attending Rare Disease Day at the University at Buffalo was both professionally meaningful and personally moving for our team at the FOXG1 Research Foundation. Events like this remind us why we do this work and how powerful true alignment can be when science, urgency, and mission come together.

From Bench to Bedside: A Story of Relentless Progress

Rare Disease Day brought together researchers, clinicians, students, and advocates committed to accelerating therapies for patients with urgent unmet needs. We were honored that FOXG1 syndrome was prominently featured throughout the day.

Our Chief Scientific Officer, Dr. Soo Lee, shared the scientific journey behind the AAV9 gene therapy developed in her lab. She walked the audience through the foundational biology, the preclinical work, and the years of focused experimentation that led to a therapy designed specifically to address the underlying cause of FOXG1 syndrome. It was inspiring to see the depth and rigor of the science and to witness how many trainees and lab members have contributed to advancing this program.

Gai Ayalon, our Chief Drug Developer, then spoke about the next critical chapter: translating an academic discovery into a clinical-stage therapeutic. Moving from bench to bedside requires manufacturing scale-up, toxicology studies, regulatory engagement, quality systems, and capital — all coordinated with precision and urgency. Gai highlighted the complexity of that transition and the deliberate steps required to prepare a gene therapy for human trials.

Finally, I had the opportunity to share a different, but equally important perspective: how this entire effort has been driven by a 501(c)(3) nonprofit organization built by families. The FOXG1 Research Foundation is not structured to maximize shareholder returns. We exist to maximize the probability of saving our children’s lives.

When incentives are aligned around patients, not profits, decision-making becomes clearer. Speed matters. Collaboration matters. Long-term impact matters. Our community of families, donors, scientists, and clinicians is unified around a singular mission: deliver a therapy to children with FOXG1 syndrome as quickly and safely as possible.

A Powerful Poster Session

One of the most illuminating parts of the day was the robust poster session. There were numerous presentations focused specifically on FOXG1 syndrome, showcasing the breadth of research underway in the Lee lab. From mechanistic studies to translational work, the posters reflected both scientific sophistication and genuine commitment.

It was particularly meaningful to hear directly from graduate students, postdoctoral fellows, and research staff about their projects. Their passion was evident. For many of them, FOXG1 is not just a gene on a slide, it is a community of children and families they have come to know.

Seeing this next generation of scientists deeply engaged in rare disease research gives us tremendous confidence in the future.

Beyond the Science

Following the event, we had dinner with the entire Lee lab. These moments away from podiums and poster boards are where relationships deepen. We shared stories about the science, about the children, about the long road to clinical trials, and about the personal motivations that drive this work.

Rare disease drug development can be isolating and difficult. But evenings like this remind us that we are not alone. We are building something together a model that demonstrates what is possible when families and scientists lock arms around a common goal.

What Rare Disease Day Represents

Rare Disease Day is more than an event. It is a reminder that small patient populations deserve the same scientific intensity and urgency as any other condition. It is proof that academic laboratories can be engines of therapeutic innovation. And it is a testament to what can happen when mission-driven nonprofits partner with world-class scientists.

For our FOXG1 families, this work is deeply personal. For the scientists, it is a calling. For our foundation, it is our responsibility.

We left Buffalo energized, grateful, and more committed than ever to advancing our AAV9 gene therapy toward clinical trials. The science is advancing. The team is aligned. And the mission has never been clearer.

We are building a path from discovery to treatment, together.