2024 marked seven years of the FOXG1 Research Foundation. We are a parent-driven, global foundation with the mission to improve the lives of every person affected by FOXG1 syndrome worldwide. We are immensely proud of the organization we have built and the resources we have made available to our community since 2017.

We kicked off 2024 with a milestone achievement: our CEO was honored as the opening speaker at the inaugural White House Rare Disease Forum. This invitation reflects our growing reputation as leaders in the rare disease space and served as a powerful platform to advocate not only for FOXG1 syndrome but for the unmet needs of all rare diseases worldwide.

We are recognized in the industry as innovators focused on three equally critical areas: FOXG1 science, FOXG1 patient data, and the FOXG1 community. From advancing our gene therapy program, which is now moving into the final stages before clinical trials, to expanding global family engagement, this year has been a testament to collective progress.

Amidst these advancements, our community deeply mourns the heartbreaking loss of five beautiful FOXG1 children in 2024. This underscores the urgency of our mission to find a cure. We are deeply grateful for the incredible team working together to achieve this vision and, most importantly, to our donors, whose generosity drives this work forward.

FOXG1 Fundraising

• Launched our first large-scale capital campaign, “Yes, They Can!”, to advance the first FOXG1 gene replacement therapy program through clinical trials.

• Raised $10 million toward our $22 million campaign goal, thanks to the incredible generosity and support of our donors.

• Surpassed our peer-to-peer crowdfunding end-of-year “Race to a Cure” goal of $250K 

FOXG1 Drug Development

• Redefined rare disease drug development with an innovative blueprint designed to significantly streamline timelines and reduce costs.

• Advanced the FOXG1 gene therapy program by:

• Selecting the lead drug candidate.

• Partnering with Charles River Labs for final stages before clinical trials including toxicology and product manufacturing.

• Securing the difficult to obtain FDA INTERACT meeting, with plans to file an Investigational New Drug (IND) application in 2025.

• Conducting “Adboard” meeting with leading neurologists to define clinical endpoints for the gene therapy trial.

• Opened the FOXG1 Research Center at the University at Buffalo (UB), featuring a cutting-edge translational lab with 20+ scientists, celebrated with a ribbon-cutting ceremony and science symposium.

• Expanded the FOXG1 Patient Biobank in collaboration with Coriell Institute for Medical Research.

FOXG1 Advocacy and Patient Support

• Our CEO and Co-founder, Nasha Fitter, was the opening speaker at the inaugural White House Rare Disease Forum, advocating for the global rare disease community.

• Hosted the largest-ever FOXG1 Syndrome Parents & Caregivers Conference in Fort Lauderdale, Florida, uniting families from 12 countries with leading FOXG1 researchers and clinicians.

• Identified 172 new FOXG1 patients worldwide.

• Grew our global volunteer network.

• Presented at and attended major conferences, including:

• American Society of Gene and Cell Therapy (ASGCT)

• American Epilepsy Society (AES)

• Digital Health & AI Innovation Summit (DHAI)

• Launched clinical research programs to better understand FOXG1 patient variants.

• Advocated for a unique ICD-10 code for FOXG1 syndrome by presenting at the ICD-10 Coordination and Maintenance Committee Meeting.

FOXG1 Gene Therapy Program Milestones

• Conduct a pre-IND meeting with the FDA.

• Complete IND-enabling toxicology studies.

• Finalize clinical research activities to ensure clinical trial readiness.

• Develop and finalize the clinical trial design and operational plan.

• File the IND application with the FDA.

Fundraising

• Raise the remaining $12 million for the “Yes, They Can!” campaign to fully fund the FOXG1 gene therapy program through trials.

• Host fundraising events worldwide through our FOXG1 Event Navigator program.

Global Growth

• Launch new affiliate FOXG1 Research Foundation chapters globally, adding to our presence in Spain, France, Australia, and Japan.

Community and Advocacy

• Expand volunteer teams across the foundation.

• Foster collaboration with families, scientists, and clinicians for ongoing advancements.

• Expand FOXG1 parent support resources, including an exciting new product to assist with daily FOXG1 caregiving.

  Thank You

Your support has been critical in making 2024 a landmark year for the FOXG1 Research Foundation. Together, we are advancing a life-changing therapy and creating a brighter future for all affected by FOXG1 syndrome.

Let’s make 2025 even more impactful!

To speak about supporting our “Yes, They Can!” campaign, please email gifts@foxg1research.org