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Voyager Therapeutics Inc is a gene therapy company focused on developing life-changing treatments for patients suffering from severe neurological diseases. It focuses on neurological diseases where an adeno-associated virus, or AAV, gene therapy approach that either increases or decreases the production of a specific protein can slow or reduce the symptoms experienced by patients, and therefore have a clinically meaningful impact. The company's gene therapy platform TRACER enables to engineer, optimize, manufacture and deliver AAV-based gene therapies that have the potential to provide durable efficacy following a single administration. Its pipeline of programs include, Anti-tau Antibody (VY-TAU01), SOD1 Silencing Gene Therapy (siRNA), FXN Gene Therapy among others.
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.