The FOXG1 Research Foundation at the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore, Maryland 2024
The FOXG1 Research Foundation is thrilled to share a recap of our participation at the American Society of Gene and Cell Therapy (ASGCT) conference in Baltimore!
This conference marked a significant milestone for our foundation, truly a "coming out party" to Pharma for our commitment to developing a gene therapy for FOXG1 syndrome.
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.”
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.
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