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ProQR Therapeutics NV is a biopharmaceutical company. It discovers and develops RNA therapeutics for patients with severe genetic rare diseases such as cystic fibrosis, Leber congenital amaurosis type 10, and dystrophic epidermolysis bullosa. The only reportable segment of the company is the discovery and development of different, RNA-based therapeutics.
Larimar Therapeutics Inc is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using its novel cell-penetrating peptide (CPP) 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.