Evaluation of ACT1 to Treat Diabetic Keratopathy

National Center For Advancing Translational Sciences

Investigators

Abstract

FirstStrings lead peptide, ACT1, is based on the C-terminal sequence of connexin43 (Cx43) and is designed to enter the cell and competitively inhibit the binding of endogenous Cx43. Cx43 plays critical roles in multiple aspects of wound healing, including spread of injury signals, extravasation of immune cells, granulation tissue formation, and fibrosis. Studies in preclinical models of efficacy show significant enhancement in corneal re-epithelialization and wound closure following ethanol-induced corneal burn injuries in diabetic rats, as compared with controls. This project will involve further development and safety studies of ACT1, a potential therapeutic compound for diabetic keratopathy. The BrIDGs team has completed PK/ADME and IND-directed toxicology studies, and developed and manufactured a clinical grade drug product, which the collaborators included in a successful IND application to initiate clinical trials. The BrIDGs team is conducting additional formulation and manufacturing work to support the ongoing clinical studies.