The FOXG1 Research Foundation is thrilled to share a recap of our participation at the 2024 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.
My FOXG1 Story, Written by Abraham Weitzman
2022 Impact Report
2022 Research Highlights (see details below)
Breakthrough gene therapy results showing rescue of FOXG1 brain structure, behavior, memory and cognition symptoms in animal models
Positive results on initial compound drug screens to identify molecules to increase FOXG1 levels; now moving forward to larger screens
Never-before understanding of FOXG1 biology uncovered from six FOXG1 patient human cell lines and mouse models
Successful testing of guide RNAs to increase FOXG1 expression with a CRISPRa Cas-9 system
Discovered ASO sequences (antisense gene therapy) to modulate FOXG1 expression; testing initiated with Creyon Bio
FOXG1 data package presented to several biotech companies with high interest
Help FOXG1 Ukrainian Family Rebuild!
The FOXG1 Research Community has helped get a FOXG1 family safely out of the warn-torn Ukraine! Now, they have to rebuild thier lives. Eva is a 3-ear-old child with the severe rare disease called FOXG1 syndrome. She suffers from seizures and is disabled. They are on thier way to safely, but they lost everything. This is how you can help. Thank you!
2021 Impact Report
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. In 2021 we raised nearly $3M. Thank you for your support!
How the Ciitizen FOXG1 Platform Drives Research and Helps Parents Manage Medical Care
Rare Disease Day 2021 : FOXG1 Syndrome
Rare Disease Day is a day to raise awareness about rare diseases like FOXG1 syndrome and their impact on patients' lives.
The research we fund at FRF not only has the potential to treat and cure FOXG1 syndrome, but provides pathways to cures for the 6,000+ other rare diseases that affect over 300 million people worldwide.
Please help us reach our goal to further cutting-edge research to find therapies for every child in the world with FOXG1 syndrome and to help pave the way for rare diseases at large.
Enjoy some Rare Disease Day 2021 FOXG1 Features
Please feel free to join us on this incredible mission! [email protected]
Thank you for your support!
Introducing - FOXG1 Research Foundation Australia!
"Inside Research with Nasha" - Repurposed Drug Screening for FOXG1
We’re kicking off a new series called “Inside Research with Nasha.”
Our FOXG1 Research strategy is to leave no stone unturned. We’re looking at every possible angle in science to find disease-modifying therapies and ultimately a cure for FOXG1 syndrome and we want to keep everyone informed along the way. We’re also taking this opportunity to really explain what so many buzz words in science mean and how they fit into the research projects we are funding.
Today, we’re very excited to talk about our small molecule drug screening projects. Nasha explains some of those buzz words that covid made mainstream, like high-throughput screening, repurposing, small molecules, and more.
You can find more information about our Research Projects, our Path to a Cure and our FOXG1 Science Team on our website.
Please follow us on social @FOXG1Research so you don’t miss any of our Inside Research updates!
What Does this New Digital Natural History Study Mean for Me?
CZI granted the FRF a half a million dollar grant to launch a next generation, machine-learning, Natural History Study. What does the new digital Natural History Study mean for FOXG1 parents? How will it help make their lives easier ? Why is it so important to help get to treatments for FOXG1 syndrome?