Acelot is treating functional protein misfolding diseases where the appropriate therapeutic mechanism is a restoration of the normal protein homeostasis. Functional misfolded proteins are found in various diseases such as ALS, FTD, Parkinson’s, diabetes and cancer. Acelot’s platform combines generative AI, molecular dynamics simulations and proprietary assays to discover small molecules that restore the homeostasis of misfolded proteins in various diseases.  We have a pipeline of hit-to-lead discovery compounds across multiple indications, along with an IND-ready candidate for ALS.  Our platform was invented by UCSB Computer Science Professor Dr. Ambuj Singh. 

Acelot’s first development candidate, ACE-2223, is a first-in-class orally bioavailable small molecule that will undergo IND-enabling studies in 2024. ACE-2223 disrupts the misfolded forms of TDP43 and restores functional TDP43. It acts upon the misfolded conformations of TDP43 found across various patient populations, including ALS, FTD and Alzheimer’s.  ACE-2223 has excellent brain penetrance and ADME properties. Acelot also has a robust discovery pipeline.

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