Path to a Cure


path.png

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 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: 1-3 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

human-sideview.png

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.

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 - $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)