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ProQR Therapeutics NV is a biopharmaceutical company. It discovers and develops RNA therapeutics for patients with severe genetic rare diseases such as cystic fibrosis, Leber congenital amaurosis type 10, and dystrophic epidermolysis bullosa. The only reportable segment of the company is the discovery and development of different, RNA-based therapeutics.
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.