Skip to main content

Small molecule and enzymatic approaches to revert aberrant phase transitions in neurological disorders

Yale Life Sciences PitchFest 2024
12/05/2024

Small molecule and enzymatic approaches to revert aberrant phase transitions in neurological disorders

We have developed MLF2-GFP, a biomarker that detects abnormal phase transitions associated with neurological diseases such as ALS, Dystonia, and FTD. These conditions currently have no cure, representing a significant unmet need in a market exceeding $2.6 billion.

Using a cell-based screening platform, we identify small molecules that influence phase behavior. Several FDA-approved drugs have shown promise in our validation studies, and we are expanding our search by screening tens of thousands of compounds to discover new candidates.

We are also utilizing MLF2 to recruit enzymes like glycosyl transferases to modify phase properties, such as increasing hydrophilicity, as a potential therapeutic approach. Our next steps include engineering MLF2 and glycosyl transferases into viral vectors to target neurons and primary cells, moving closer to clinical applications.

Download Pitch Presentation