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Editas Medicine Inc is a clinical-stage genome editing company dedicated to developing potentially transformative genomic medicines to treat a broad range of serious diseases. The company focuses on developing a proprietary genome editing platform based on clustered, regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9 technology). It has developed CRISPR/Cas9 technology which uses a protein-RNA (ribonucleic acid) complex composed of the Cas9 enzyme bound to a guide RNA molecule designed to recognize a DNA (deoxyribonucleic acid) sequence that requires repair. The company is engaged in developing and commercializing genome editing technology.
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.