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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.
uniQure NV is a gene therapy company. It develops treatments and platforms for patients suffering from genetic and other devastating diseases. Its products and services are focused on hemophilia, Huntington's disease, and cardiovascular diseases. The company is focused on the development of the pipeline of gene therapies with the collaboration of Bristol Myers Squibb for cardiovascular diseases.