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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.
Protalix BioTherapeutics Inc is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins based on its proprietary ProCellEx protein expression system or ProCellEx. It has developed taliglucerase alfa for the treatment of Gaucher disease. The company's products also treat diseases like Fabry disease and cystic fibrosis. The company's primary sources of revenue include its sales of BioManguinhos alfataliglicerase in Brazil and of drug substances to Pfizer under its Amended Pfizer Agreement. The company's pipeline consists of ELFABRIO, Uricase, and Long Acting DNase I.