Larimar Therapeutics

Small Molecules

Larimar Therapeutics is developing therapies for rare genetic diseases that currently have no effective treatments. Genetic mutations can cause cells to have low levels of a protein which leads to disease. Using their proprietary protein replacement therapy platform, Larimar is developing disease modifying therapies that deliver missing proteins directly to cells that need it. Larimar’s lead candidate, CTI-1601, is currently being evaluated in Phase 1 clinical trials for Friedreich’s ataxia. Friedreich’s ataxia patients have low levels of endogenous Frataxin, a protein that functions in the mitochondria. CTI-1601 is a fusion protein that contains Frataxin and crosses the cell membrane into the cytoplasm. CTI-0601 also contains information to enter the mitochondria, where it is needed.