Drug Repurposing

Team Leader:

Benjamin Prosser, PhD

Benjamin Prosser, PhD

ASO Leadership Drug Repurposing Leadership Leadership ASO CRISPR Drug Repurposing Gene Replacement
repurposing scheme

ENDD has partnered with Panorama Medicine to repurpose FDA-approved compounds for both STXBP1 and SYNGAP1 disorders. After screening thousands of compounds, we have identified several FDA-approved lead candidates that are blood-brain barrier penetrant and can upregulate target gene expression. We are currently testing them in our animal models for efficacy, phenotype rescue, and toxicology.