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CRISPR Therapeutics is a leading gene-editing company focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. Our multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Our lead programs in beta-thalassemia and sickle cell disease have advanced to IND/CTA-enabling studies with a CTA filing planned by the end of 2017, and we are advancing additional programs in ex vivo and in vivo disease areas. In addition to our fully-owned programs, our strategic collaborations with Bayer AG and Vertex Pharmaceuticals expand our portfolio and enable us with unique capabilities. Through our private financings, partnerships, and IPO we have raised >$400M to fund and accelerate our portfolio. We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. Our company is headquartered in Zug, Switzerland with R&D operations in Cambridge, Massachusetts, USA and some business operations in London, United Kingdom.
CombiMatrix Corporation is a Irvine, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs for serious ocular and rare diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of its lead indication, wet age-related macular degeneration.
Surrozen is a biotechnology company focused on discovering and developing novel regenerative medicines with a focus on unlocking the powerful self-renewal properties of the body through specific control of the Wnt signaling pathway.
Synthorx Inc. is a biotechnology company using synthetic biology to discover and develop novel protein therapeutics. Synthorx`s expanded genetic alphabet platform has the unique ability to drive the site-specific incorporation of multiple synthetic amino acids into proteins thereby tuning receptor specificity important for improving efficacy and safety. In addition, their engineered organisms provide the ability to manufacture these improved proteins, called Synthorins, with the required fidelity and yield. The unique Synthorx platform expands the chemical and structural repertoire of protein therapeutics and uncovers new ways to modulate pharmacological properties of biologics, not possible with other technologies. The company was founded based on important discoveries in Dr. Floyd Romesberg`s lab at The Scripps Research Institute in conjunction with Avalon Ventures, which housed Synthorx in its incubator, COI Pharmaceuticals, Inc. The company is headquartered in La Jolla, Calif.