Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Headquartered in Bala Cynwyd, Pennsylvania, the company utilizes a novel cell-penetrating peptide (CPP) technology platform to deliver therapeutic molecules to intracellular targets. Its lead product candidate, nomlabofusp (CTI-1601), is a subcutaneously administered recombinant fusion protein intended to deliver human frataxin (FXN) to the mitochondria of patients with Friedreich's ataxia (FA). FA is a rare, progressive, and fatal genetic disease characterized by insufficient production of FXN, for which nomlabofusp has received Rare Pediatric Disease, Fast Track, and Orphan Drug designations from the FDA. Beyond FA, Larimar plans to leverage its proprietary intracellular delivery platform to design additional fusion proteins targeting other rare diseases caused by deficiencies in intracellular bioactive compounds. The company assumed its current form in 2020 following a reverse merger between Chondrial Therapeutics and Zafgen, Inc.
