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/

FRF Appoints Chief Drug Development Officer, Dr. Gai Ayalon

We’re thrilled to announce the appointment of Dr. Gai Ayalon as the Chief Drug Development Officer of the FOXG1 Research Foundation (FRF).

Dr. Ayalon is a distinguished neuroscientist and drug developer, who over many years led teams and spearheaded drug development programs for neurological diseases including rare neurodevelopmental disorders, spanning the discovery, translation and clinical phases. He joins our foundation at a critical and pivotal juncture as we enter Phase Four on our Path to a Cure, taking programs through safety studies and clinical trials. 

We have successfully accomplished Phase One through Three on our Path to a Cure: building disease models, studying these models, and testing gene therapies, ASO's, CRISPRa and saRNA therapies on our models. These early experiments have shown that we can upregulate FOXG1 gene and protein levels, we can do so safely, and we can rescue key symptoms in animals such as motor function, cognition and corpus callosum degeneration. Dr. Ayalon will now lead this next phase by taking our therapeutic programs, one by one, to clinical trials. In order to do this we will be performing activities such as vector optimization, exploratory toxicology, GLP manufacturing and toxicology, and completing regulatory filings. 

Dr. Ayalon previously worked at Ultragenyx Pharmaceutical, where he was the Project Team Leader of programs for pediatric neurodevelopmental disorders such as Angelman syndrome. Prior to Ultragenyx, Dr. Ayalon was a scientist at Genentech, in the neuroscience department. At Genentech he led drug discovery programs and teams focused on immunotherapeutic approaches to neurodegenerative diseases. 

Most recently, Dr. Ayalon was Vice President, Head of the Portfolio and Program Management group at Neumora Therapeutics, where he also led clinical stage programs for neuropsychiatric disorders. Dr. Ayalon received his Ph.D. from the Hebrew University Medical School in Jerusalem, Israel, and conducted his postdoctoral research at the Howard Hughes Medical Institute at the Duke University Medical Center.  

In his new role as Chief Drug Development Officer, Dr. Ayalon will navigate the FOXG1 Research Foundation through clinical drug development, working closely with Dr. Soo-Kyung Lee’s lab at the University at Buffalo, as well as the FOXG1 Scientific Consortium of labs, and our global biopharma and clinical partners.  

We cannot be more optimistic about joining hands with Dr. Ayalon for this next chapter in our journey to greatly improve the lives of every patient and the families affected by FOXG1 syndrome.