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Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.
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.