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GenSight Biologics is is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics` pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics` lead product candidate, GS010, is in Phase III trials in Leber`s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics` product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Five Prime Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of protein therapeutics that block cancer and inflammatory disease processes. The company`s product candidates include FP-1039/GSK3052230, a protein therapeutic is in PhaseIb clinical trials for trapping and neutralizing cancer-promoting fibroblast growth factors (FGFs) involved in cancer cell proliferation and new blood vessel formation; FPA008, an antibody that inhibits colony stimulating factor-1 receptor; and FPA144 is an antibody for inhibiting FGF receptor 2b, as well as to treat patients with gastric cancer and potentially other solid tumors. It has collaboration and license agreements with GlaxoSmithKline LLC, Glaxo Group Limited, GSK-HGS, Pfizer Inc., and UCB Pharma S.A. The company was founded in 2001 and is headquartered in South San Francisco, California.
Precision BioSciences is a publicly held biotechnology company dedicated to improving life through its next generation gene editing technology, ARCUS. Precision`s cell therapy, gene therapy, and agriculture programs are based on the proprietary ARCUS gene editing platform, which features coordinated target site binding and DNA cutting to ensure precise edits. Our team includes pioneers in genome engineering, biotech business leaders, and a growing staff of committed Precisioneers. We work together in our beautifully designed, award-winning space in vibrant downtown Durham, NC; a place with great restaurants and museums as well as fantastic housing and schools. Precision provides all employees a sense of purpose, thoughtfully designed laboratory and office spaces, and opportunities to train and grow – not to mention plentifully stocked kitchens and fantastic benefits. At Precision BioSciences, we are excited to work together toward improving lives by realizing the potential of gene editing.
The Boston Biomedical Research Institute (BBRI) is dedicated to basic biomedical research to promote the understanding, treatment and prevention of specific human diseases. The areas of investigation concern the structure and function of muscle proteins,
Loxo Oncology is committed to the discovery, development, and commercialization of targeted cancer therapies with best-in-class potential. Our diverse pipeline reflects the convergence of proven therapeutic technologies with emerging insights into the underlying susceptibilities of cancer and drug resistance. It is an exciting time to develop cancer drugs. Diagnostic methods are yielding strong clues about which cancer features are actionable, “driver alterations,” in contrast to those merely along for the ride as “passenger alterations.” In the last five years, these insights have transformed the standard of care in melanoma, lung cancer and other cancers. Picking a target is just the first step in building a cancer drug. Good chemists build good drugs. The physical properties of a compound have much to do with its ultimate success or failure. Is it stable? Is it soluble? Is it absorbed? Does it reach its target? Does it engage the target? Does it cause toxicity elsewhere in the body before it achieves maximum efficacy in the cancer? In our opinion, drugs that achieve excellent exposure in the body and are highly specific for their intended targets have the highest chances of success. Once an attractive target is selected and a drug with best-in-class properties is constructed, setting up the right clinical development plan is of utmost importance. Our goal is to generate a clinical path that reflects the underlying scientific hypothesis that made the drug interesting in the first place. This often means developing the drug in a population of patients whose tumors possess a specific vulnerability targeted by the drug. Loxo Oncology was built around a team of full-time professionals and engaged scientific advisors who are aligned in choosing the most actionable targets, and pursuing them through disciplined clinical trial approaches. Our license and collaboration agreement with Array Biopharma and our experienced team allow us to construct drugs with best-in-class properties from the ground up.