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2022 Impact Report

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