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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Taysha Gene Therapies Takes on FOXG1 Syndrome!

October 21, 2020

Dear FOXG1 Community,

As you may have learned, Taysha Gene Therapies is a new company that launched at the end of April focusing on developing gene therapies for central nervous system disorders.

Our Founder, President and CEO RA Session II became excited about the research program prior to joining Taysha after learning more about the high unmet medical needs associated with the disease. When RA founded Taysha, he recognized how certain technological gene therapy approaches available at our company, specifically those able to regulate re-expression of protein, could enable the team to identify a unique method of treating the cause of this disease in a regulated manner. It is now our pleasure to introduce the FOXG1 Community to Taysha Gene Therapies.

Taysha is a word in the Caddo Native American language meaning “ally” or “friend,” and when translated, also means “Texas.” Between our UT Southwestern collaboration and patient advocacy partnerships, we hope to be an ally to the rare disease community, including the FOXG1 community.

Our focus is gene therapy, which is a highly innovative approach to treating genetic diseases. We are working hand-in-hand with our collaborators at UT Southwestern collaborators, who are well-known gene therapy experts, medical experts in FOXG1 Syndrome, regulatory agencies and, importantly, patient groups every step of the way. We are truly embracing an integrated approach—one that will help get new medicines in the hands of physicians treating children affected by FOXG1 Syndrome in the most efficient, effective way possible.

We have a lot of work ahead of us but are encouraged by the strength of the FOXG1 Research Foundation that has been built to achieve this goal. By working together, our efforts will help bring gene therapy to the FOXG1 Community.

Warm regards,

The Taysha Team