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Larimar Therapeutics Inc is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using novel cell-penetrating peptide technology platform. Its lead product candidate, CTI-1601, is a subcutaneously administered, recombinant fusion protein intended to deliver human frataxin (FXN) an essential protein, to the mitochondria of patients with Friedreich's ataxia.
Regenxbio Inc is a biotechnology company. Its main activity is the development and commercialization of recombinant adeno associated virus gene therapy to correct an underlying genetic defect. The diseases that Regenxbio platform targets are metabolic (homozygous familial hypercholesterolemia), neurodegenerative conditions (mucopolysaccharidosis), and retinal diseases (wet age-related macular degeneration, X-linked retinitis pigmentosa). The company derives the majority of its revenue from the United States.