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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, nomlabofusp, 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 an unmet medical need for treatments designed to address the root cause of the disease. Nomlabofusp has been granted Rare Pediatric Disease designation, Fast Track designation, orphan drug status by the U.S. Food and Drug Administration (FDA) and the the European Medicines Agency (EMA) and a PRIME designation by the EMA. Larimar’s nomlabofusp development program was also selected by the FDA to participate in its START (Support for clinical Trials Advancing Rare Disease Therapeutics) pilot program, which is designed to accelerate development of novel therapies intended to address an unmet medical need for rare diseases. 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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