We are so excited to deliver this Research Update during FOXG1 Awareness Month!
Two Biotechs are investing in FOXG1 syndrome!
We’re moving into Phase Two and Phase Three of our Path to a Cure!
1. We have Multiple Biotech Investments in FOXG1!
When we launched The FOXG1 Research Foundation in 2017, our goal was to develop the science needed for biotech companies to take us on. As a result of our investments made in developing patient-derived stem cell models, animal models, developing rich patient data, and having amazing community support, we were able to attract the world class talents of Taysha Gene Therapies and Creyon Bio to work on FOXG1 syndrome!
Taysha Gene Therapies: We’re thrilled to announce that Taysha Gene Therapies is developing TSHA-117 for FOXG1 Syndrome. Because the condition requires replacement of dose-sensitive genes, one investigational approach the company plans to explore is using its novel miRNA target panel, which is designed to enable the product candidate to maintain safe transgene expression levels in the brain.
Taysha is focused on developing AAV-based gene therapies specifically for monogenic CNS diseases. Dr. Sarah Sinnett at UT Southwestern leads the FOXG1 syndrome pre-clinical program. In addition, Dr. Sinnett and Dr. Steven Gray, also at UT Southwestern, are working on developing novel approaches for Rett syndrome. Read the announcement here.
Creyon Bio for ASO therapy: ASOs or “antisense oligonucleotides” are an exciting disease-modifying therapeutic approach showing real results in the Angelman and Batten Disease communities.
We are honored and excited to announce a partnership with Creyon Bio to explore and hopefully advance new oligonucleotide based therapeutics for the treatment of FOXG1 syndrome. Creyon Bio is focused on making new gene-centric medicines affordable and rapidly available to patients globally. Creyon Bio is adopting advances in computational chemistry, biophysics and ML/AI to build a scalable platform that will transform the way oligonucleotide-based medicines (OBMs) are created. The Creyon team brings decades of experience in quantitative biology, genomics and OBM development and is excited to forge a strong collaboration with the FRF Science Team, including Dr. Alysson Muotri, Dr. Soo-Kyung Lee and others.
NOTE: The collaboration described herein is subject to the negotiation and execution of definitive legal documentation by FOXG1 Research and Creyon.
2. We’re right on trajectory along the Path to a Cure!
Thanks to all of your help, we were able to create the necessary models to understand FOXG1 syndrome as planned. We have successfully:
funded and developed FOXG1-specific mouse models
created a biobank of FOXG1 patient skin and blood samples, which we were able to turn into Induced Pluripotent Stem Cells and Mini Brain Organoids.
This took a long time, and we have all been patiently (sort of) waiting to get to the fun part.
Now, we start Phase 2 and 3: Studying and Screening Drugs and Therapies on these models.
What are we studying?
How FOXG1 mutations affect brain circuits, different brain cells and human biology. What can we target to cure our children?
What downstream genes are affected by FOXG1; are any of them fixable?
What biomarkers can we find to test drugs against? Can we connect these biomarkers to clinical endpoints in our children?
When we screen drugs, how do they affect protein expression, functional assays and downstream targets?
What are we screening?
Small molecule drugs. Here’s a success story in a similar disease.
Antisense therapies. Here’s a success story in a similar disease.
Gene therapies. Here’s a success story in a similar disease.
We are also converting Astrocytes to Neurons:
In this breakthrough discovery, scientists at UCSD found a fairly simple way to convert astrocytes into neurons. These scientists were further stunned to see that when this treatment was given to Parkinson’s mice, movement disorder symptoms reversed and did not come back for the entire life of these mice. We know that FOXG1 syndrome leads to neuronal death and thus increasing the amount of neurons could reverse major symptoms. Dr Alysson Muotri, whose lab is luckily at UCSD, will be collaborating with these scientists to conduct similar experiments on FOXG1 astrocytes and brain organoids.
Our goal in 2021 is to find both short-term drugs we can give our children today, as well as longer term solutions like ASO’s, gene therapies and other novel and emerging therapies.
We’ve assembled a top tier FOXG1 Science Team to collaborate to achieve these goals:
High Throughput Screening
Houart Lab, King's College - High throughput screening of small molecules on zebrafish
Stemonix - A contract research organization conducting high throughput screening of small molecules, vitamins and herbs on neurospheres
Bedwell lab, University of Alabama - Screening ~200 primary, validated, non-cytotoxic hits to suppress nonsense mutations in cell lines
Targeted Screening (low-throughput)
Muotri Lab, UCSD - Screening of small molecules, antisense therapies, RNA therapies and gene therapies on neurospheres, IPS lines and organoids. Also characterizing/phenotyping these models
Lee lab, University at Buffalo - Screening of small molecules, antisense therapies, RNA therapies and gene therapies on mouse models. Also characterizing/phenotyping these models
Malamaci lab, SISSA - Developing RNA therapies for screening on IPS lines and mouse models
Creyon Bio - Developing targeted antisense therapies to screen on organoids and mouse models
Meyer lab, Nationwide Childrens - Developing gene therapy approaches and new in vitro model systems; screening small molecules and looking at the mechanisms (protein expression, downstream targets) on IPS lines and mouse models
The collaboration within this esteemed team of scientists and labs will enable us to find a basket of solutions to cure FOXG1 syndrome.
WE NEED ALL HANDS ON DECK TO FUND THIS SCIENCE NOW!
We’re often told that we have accomplished more in these last three years than most organizations do in ten. As a community, we all pulled together and raised nearly $2 million to launch the critical Phase One projects. We couldn’t have done this without everyone’s fundraising support.
Now, it’s time for our next push - we need to raise another $1.5 million in the next six months to screen and test potential therapies. We need all hands on deck to come together and move mountains for our children. Here are a few things you can do:
Please donate here.
Ask your friends and family to help us reach this important goal
We’ve added the option to Donate Stock
Buy your holiday presents at our store (more items coming very soon!).
Create a Facebook fundraiser. Let followers know we need all hands on deck to get to clinical trials!
If you can, offer to MATCH donations up to an amount.
Buy toys and all special needs items for children through our Amazon Affiliate program on our website.
Go to smile.amazon.com every time you make any purchases on Amazon and set FOXG1 Research Foundation. Tell your friends and family to do this too!
Thank you for supporting us on this journey.
And stay tuned as we have more exciting announcements coming this month!!
With much gratitude,
The FOXG1 Research Foundation Team