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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.
Silence Therapeutics PLC is a biotechnology company focused on discovering and developing novel molecules incorporating short-interfering ribonucleic acid or siRNA. Using its mRNAi GOLD platform, the company has generated siRNA product candidates both for internal development pipeline as well as for out-licensed programs with third-party collaborators. Its wholly owned pipeline is focused on three therapeutic areas: cardiovascular disease, hematology, and rare diseases. The different product candidates in its pipeline are Divesiran (SLN124), a siRNA product candidate that has the potential to treat several hematological disorders; Zerlasiran (SLN360), being developed as a potential treatment to reduce cardiovascular issues; SLN-312; SLN-548; and multiple other programs.