Larimar is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using its novel cell penetrating peptide technology platform. Larimar’s lead product candidate, CTI-1601, is a recombinant fusion protein intended to deliver human frataxin (FXN), an essential protein, to the mitochondria of patients with Friedreich’s Ataxia (FA). FA is a rare, progressive and fatal disease in which patients are unable to produce enough FXN due to a genetic abnormality and for which there is currently no effective therapy. Larimar has received orphan drug status, fast track designation, and rare pediatric disease designation from the FDA for CTI-1601, which is currently being evaluated in a Phase 1 clinical trial. Larimar Therapeutics also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.
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