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Taysha Gene Therapies Inc is a patient-centric gene therapy company to eradicate monogenic CNS disease. It is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the CNS in both rare and large patient populations. The company develops and commercializes transformative gene therapy treatments. Its product pipeline includes TSHA-102 which is a is a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome.
Silence Therapeutics PLC is a biotechnology company focused on discovering and developing novel molecules incorporating short-interfering ribonucleic acid. its pipeline products are; SLN360 therapy designed to temporarily block a specific gene's message that would otherwise trigger an unwanted effect. By silencing the LPA gene, the levels of Lp(a) are lowered, which in turn is expected to lower the risks of heart diseases, heart attacks, and strokes, and SLN124 therapy aims to temporarily silence TMPRSS6, a gene that prevents the liver from producing a particular hormone that controls iron levels in the body hepcidin. As hepcidin increases, iron levels in the blood are expected to decrease, which may increase the production of healthy red blood cells, thereby reducing anemia.