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

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