Path to a Cure


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This is the roadmap to find a cure for mutations to the FOXG1 gene. This work is critical to understand and cure autism and many brain disorders. We have the leading neuroscientists in the world working with us 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 Funding Goals page.

PHASE ONE: 1-2 YEARS

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
  • Identify therapies and evaluate efficacy

Identify and Vet Gene Therapies

  • Test various gene therapies to see if FOXG1 gene restoration alleviates symptoms
  • Learn which time points effective for treatment
  • Test safety of therapies in animals 
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Gene therapies will insert a healthy FOXG1 gene or edit parts of the FOXG1 gene directly in our children’s brains.

 

Identify and Vet Small Molecule and Other Therapies

  •   Screen existing FDA approved small molecules on identified biological pathways that can modulated

The first early study done at University of Massachusetts using RNA gene therapy in a FOXG1 mouse was successful.

saRNA- and AAV-Foxg1-mediated approaches to boost endogenous expression
CRISPR/Cas9 editing as a tool to correct FOXG1 mutations in neurons
RNAi as a tool to silence dysfunctional protein
 

PHASE TWO: 1-2 years

Pre-clinical testing of successful therapies

  • Important feasibility, iterative testing and drug safety data are collected 

PHASE THREE: 1-5 years        

Human Clinical Trials

 


PHASE FOUR: TBD

FDA Approved* therapy for alleviating symptoms of FOXG1 mutations

(*and other agencies)