Path to a Cure
This is the roadmap to find a cure for mutations to the FOXG1 gene. This work is critical to understand autism and many brain disorders. We are working with the leading neuroscientists in the world in each category. Our path to a cure follows four phases. We are currently in Phase One.
See the Phase One projects we have underway and those we are raising money to fund on our Research Projects page.
PHASE ONE: 1-3 YEARS - $3M
Generate and Characterize Assets for Testing
- Build FOXG1 mouse models and patient derived stem cells (iPS cells)
- Study these models to understand the impact of FOXG1 on the brain and body
- Learn if FOXG1 mutations can be "fixed" post natal
Identify and Vet Gene Therapies
- Test various gene therapies to see if FOXG1 gene restoration alleviates symptoms
- Learn which time points are effective for treatment
- Identify successful therapies and test efficacy
- As technology advances, we will continue to consider new therapies
Identify and Vet Small Molecule and Other Therapies
- Screen existing FDA approved small molecules on identified biological pathways that can modulated
FOXG1 is an excellent candidate for gene therapy. Learn Why
Types of Gene Therapy
• Editing at the DNA level (CRISPR)
• Editing at the RNA level
• Activating / Inserting a healthy copy of the FOXG1 gene (saRNA, RNAi)
The first early study done at University of Massachusetts using RNA gene therapy in a FOXG1 mouse was successful.
Gene Therapy through AAV to boost protein expression"
CRISPR/Cas9 editing as a tool to correct mutations in neurons
RNAi as a tool to silence dysfunctional protein
PHASE TWO: 1-2 years - $7M per trial
Pre-clinical testing of successful therapies
• Important feasibility, iterative testing and drug safety data are collected
PHASE THREE: 1-5 years - Biotech investment
Human Clinical Trials
PHASE FOUR: TBD
FDA Approved* therapy for alleviating symptoms of FOXG1 mutations (*and other agencies)