FOXG1 Research Foundation ASGCT Recap -> On Track to a FOXG1 Gene Therapy

The FOXG1 Research Foundation recaps our participation at the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore!

This conference marked a significant milestone for our foundation, an introduction to Pharma for our commitment to developing a gene therapy for FOXG1 syndrome.

The FOXG1 Research Foundation at the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore, Maryland 2024

Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA

A Pivotal Conversation about FOXG1 syndrome with the FDA

One of the highlights of the conference was the opportunity to speak with Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA. We discussed the FOXG1 Research Foundation's gene therapy program and the upcoming steps in our journey. This conversation was a significant milestone, paving the way for a productive collaboration with the FDA as we move forward.

Setting the Stage for FOXG1 Gene Therapy IND Approval

We are thrilled to announce that we have officially submitted our request for an INTERACT meeting with the FDA. This meeting is a crucial step in the Investigational New Drug (IND) application process, which allows us to initiate clinical trials for our gene therapy treatment. This marks the official beginning of our IND enabling journey!

FOXG1 Research Center of Excellence at the University at Buffalo poster: AAV9-FOXG1, a gene therapy vector, in a mouse model of FOXG1 syndrome

Groundbreaking Posters on FOXG1 Research Presented

The conference also provided a platform for showcasing the latest advancements in FOXG1 research. The Lee lab from the University of Buffalo presented a captivating poster on their research using AAV9-FOXG1, a gene therapy vector, in a mouse model of FOXG1 syndrome. Their findings suggest the potential of this approach in rescuing corpus callosum agenesis and other brain deficits associated with the syndrome. This research aligns perfectly with our mission and adds valuable data to the growing body of evidence supporting gene therapy as a viable treatment option.

New Findings on the Epidemiology of FOXG1 Syndrome

Another impactful presentation came from our advisor, Karen Malone, who addressed the critical challenge of describing the unmet need for treatments in rare diseases like FOXG1 syndrome. Dr. Mallone’s poster showcased innovative strategies for effectively communicating the urgency and importance of developing therapies for this patient population. The conclusion is that FOXg1 syndrome is more prevalent than currently known:

“FOXG1 Syndrome was previously considered an ultra-rare indication potentially occurring in ~1 per million children. Our analysis based on genetic testing demonstrates the FOXG1 patient population is expected to be approximately one third the size of MECP2 patients largely associated with Rett Disease.”

Estimating Patient Prevalence for Complex Neurodevelopmental Disorders: The Emerging Face of FOXG1 Syndrome

FOXG1 Team Building Connections and Advancing the Field

The ASGCT conference was not only about sharing our progress, but also about forging valuable connections. We had the privilege of interacting with leading figures in the gene therapy field, fostering collaboration and knowledge exchange. 

The momentum gained at the ASGCT conference is truly inspiring. We at the FOXG1 Research Foundation are energized and focused on driving successful genetic therapies for FOXG1 syndrome.

Watch FOXG1 Research Foundation’s CEO Nasha Fitter’s ASGCT take-aways…

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FOXG1 Research Foundation 2024 Q2 Research & Development Update

The FOXG1 Research Foundation is pleased to share our 2024 Q2 update on FOXG1 Research and Development from our Chief Scientific Officer, Dr. Soo-Kyung Lee and our Chief Drug Development Officer, Dr. Gai Ayalon.

FOXG1 Research:

• FOXG1 Syndrome Epidemiology manuscripts under review 

• FOXG1 mouse model manuscript is under review

• Three abstracts submitted for the ASGCT meeting in May, which will be attended by member of the FOXG1 Research Foundation Scientific, Clinical, and Executive team

• Important preclinical study initiated to further support the choice of the AAV9 drug candidate to progress to the next stage of toxicology studies and manufacturing work

• Initiation of preclinical studies aimed at the characterization of function of FOXG1 in brains of juvenile, young adult, and adult animals

• Investigation of FOXG1 syndrome pathogenesis in human neurons derived from patient cells (thank you to the families that provided patient cells)

• Refined the ASO and small molecule drug repurposing efforts with focus on enhancing the screening platforms

• Continue to support and collaborate with external academic labs with focus on projects with high translational potential

FOXG1 Translation and Development:

• Secured IRB approval for the expanded FOXG1 Coriell Biobank, in final steps of contracting and initiation of the new repository

• Completion of enrollment and sample collection for the Combined Brain biomarker initiative (thank you to the families that participated)

• FOXG1 Disease Concept Model project to inform future trial design and regulatory interactions is in progress (thank you to the families that are participating)

• Launched the development core team which is developing in detail the toxicology, manufacturing and regulatory plans for the AAV9 gene therapy program

• Preparing the data package and meeting request submission for our first interaction with FDA on the AAV9 gene therapy program, the first of several FDA engagements that are planned for 2024 to advance the program through IND-enabling studies

• Initiated a collaboration with other Patient Advocacy Groups (PAGs) to evaluate digital biomarker devices for use in future clinical trials

• In the designing phase of a clinical research study to characterize mosaicism in FS, which could help inform the design of future clinical trials

• Planning the 2024 FOXG1 Research Foundation private science symposium with a focus on translational programs and clinical trial readiness

• FOXG1 Research Foundation Australia is preparing for clinical trial readiness with the Sydney Childrens Hospital Network and the Rare Care Centre in Perth, Western Australia

FOXG1 Research Foundation Chief Scientific Officer, Dr. Soo-Kyung Lee and Chief Drug Development Officer, Dr. Gai Ayalon at the FOXG1 Research Center at the University at Buffalo/