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Editas Medicine Inc is a clinical-stage genome editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. The company focuses on developing a proprietary genome editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9 technology). It has developed CRISPR/Cas9 technology which uses a protein-RNA (ribonucleic acid) complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a DNA (deoxyribonucleic acid) sequence that requires repair. The company is engaged in developing and commercializing genome editing technology.
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.