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Hawaii Biotech is a Aiea, HI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Xpogen is a Newton Center, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
BioMarin is a global pharmaceutical company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We remain steadfast to our original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options and are usually ignored. Time is critical to patients with rare diseases, and we strive to quickly develop important therapies for them. The efficiency and speed of our research, development, manufacturing, and commercial efforts is at the heart of our ability to urgently deliver therapies. Our track record of developing and commercializing new treatments has been significantly faster than the industry average and is a remarkable accomplishment that is ingrained in our culture. The company`s product portfolio comprises five marketed products and multiple clinical and pre-clinical product candidates.
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.
VelosBio Inc. is a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, ROR1-directed therapeutics to transform the lives of patients with cancer. Its lead candidate, VLS-101, is a ROR1-directed ADC being developed for patients with hematologic and solid tumor malignancies. The company is developing a broad pipeline of therapeutics targeting ROR1 in oncologic indications with high unmet medical need.