The Impact of Donors Support for the FOXG1 Research Foundation

The FOXG1 Research Foundation is dedicated to driving science towards therapeutics and ultimately a cure for every person in the world affected with FOXG1 syndrome. While accelerating research, we are also focused on advocating for and supporting families along this difficult journey. We cannot do this without you. Thank you for your support!

We have raised nearly $3M from November 2020 - November 2021

How Funds Were Spent in 2021:

• Funded renown gene therapist Dr. Kathrin Meyer at Nationwide Children’s Hospital; testing of several gene therapies underway in collaboration with the FOXG1 Center of Excellence at University at Buffalo

• Hired Director of Science, Dr. Hourinaz Behesti, from Rockefeller University in New York

• Joined the Rarebase “Function” Neuroscience Platform to screen FDA approved drugs 

• Continued testing of FDA approved drugs on FOXG1 zebrafish models at Kings College, London

• Funded UC Davis’s Fink lab to develop a CRISPR Activation therapy

• Funded another year at the FOXG1 Center of Excellence, led by the world’s scientific expert on FOXG1, Dr. Soo-Kyung Lee; will be publishing her first groundbreaking scientific data this year

• Developed and filed provisional patent for first antisense oligonucleotide therapy; therapy in phase two of testing

• Continued funding of Dr. Alysson Muotri at UCSD to develop FOXG1 3D brain organoids for drug testing

• Expanded FOXG1 global patient registry to new languages and introduced a series of FDA validated surveys

• Launched innovative machine learning platform with Ciitizen/Invitae to collect and extract clinical data to correlate variants seen in patients to their clinical symptoms (natural history study)

• Officially hired Executive Director, Nicole Johnson, mom to Josie, and FRF co-founder

• Funded group of leading neurologists from Harvard and CHOP to analyze data from patient registry and Ciitizen natural history. Four publications are planned for 2022, which will catapult biotech interest and understanding of FOXG1 syndrome

• Created two new mouse models and six new stem cell lines 

FOXG1 Research Foundation Accomplishments: 

• Partnered with four biotech companies to include FOXG1 in target list

• Became a Chan Zuckerberg Initiative “Rare As One” partner and mentor

• Gathered the most comprehensive dataset spanning more than 12 years per patient from the most FOXG1 patients ever studied

• Identified multiple sequences to upregulate FOXG1 gene and protein expression

• Nominated for Global Genes Champions of Hope Founders Award 2021

• Invited as a speaker to a plethora of science podcasts, radio interviews, and conferences

• Partnered with the Autism BrainNet to help promote research on postmortem brain tissue to advance the knowledge of FOXG1 and for identifying targets for treatments

• Identified as thought leaders and mentors in the rare disease space

Fundraising Goal: $10M to drive the science to clinical drug development

• Accelerate each project by funding additional post-docs 

• Validate and bring forward to clinical trials successful antisense oligonucleotide and gene therapy proof-of-concepts  

• Fund further understanding of FOXG1 symptoms, such as electrophysiology characterization in mouse models

• Fund a phenotypic drug screen with Rumi scientific

• Purchase larger sets of FDA validated drugs for testing on our various platforms

• Build additional assets, such as conditional rescue zebrafish model

• Continue to expand upon patient registry and natural history study work

• Reserves for clinical drug development 

• Create FOXG1 Caregivers Fund for support grants