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Our vision is to transform drug discovery from a slow, sequential, and high-failure process into a rapid, integrated, and novel computational platform to accelerate drug discovery.
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.
We work at the intersection of biology, engineering, and design, developing technologies that emulate human biology. Our products are enabling a new era of precision medicine and the development of applications for personalized health. Our Human Emulation System, which uses Organs-on-Chips technology, is being used by industry, government, and academia to understand how different diseases, medicines, chemicals and foods affect human health. The system offers researchers a new standard for predicting human response — with greater precision and control than today`s cell culture or animal-based experimental methods. We are also leading a bold, new precision medicine initiative with our clinical partners. Named "Patient-on-a-Chip," the program is developing personalized chips with patients` own cells. This initiative will help transform the way each of us understands our own bodies and manages our own health. Research conducted using our Organ-Chips has been published in high-impact scientific journals, including Science. In addition, Organ-Chips have been acquired by the Museum of Modern Art (MoMA) in New York for their permanent collection, and have also been awarded Product Design of the Year 2015 by London`s Design Museum. Our founding team pioneered the original Organs-on-Chips technology during their residency within the Wyss Institute for Biologically Inspired Engineering at Harvard University.
MBio Diagnostics is now LightDeck Diagnostics! LightDeck Diagnostics believes in a new approach to healthcare, where fast, accurate, simple, low-cost diagnostic tests will be run wherever and whenever they are needed, without compromise.
Applied Molecular Therapeutics (AMT), a public biopharmaceutical company based in South San Francisco, California, is committed to developing novel, oral biological therapeutics to treat severe autoimmune, metabolic and inflammatory diseases. AMT is leveraging its propriety technology platforms to create gastrointestinal (GI)-select therapeutics that harness naturally occurring transport and targeting mechanisms to cross the protective barrier of the intestinal epithelium (IE). Once across the IE, the therapeutics gain privileged access to the immune cell-rich environment of the GI tissue as well as the hepatic portal system and downstream systemic circulation. With this privileged access, AMT`s goal is to develop transformative new oral therapeutic treatment options that offer patients greater efficacy and tolerability than available with today`s therapeutics. AMT has a robust pipeline of oral biologic product opportunities, including AMT-101 which is currently in Phase 1b clinical development for the treatment of adults with ulcerative colitis (UC). AMT-101 is a novel, gut-selective, investigational oral biologic fusion protein of interleukin 10 (IL-10), an anti-inflammatory cytokine. Derived from AMT`s proprietary platforms, AMT-101 is engineered to cross the selective barrier of the IE and enhance localized IL-10 directly within the immune-cell rich environment of GI tissue where up to 75 percent of immune cells reside. This direct targeting of the immune system creates the potential for AMT-101 to address, at its point of origin, the immune dysregulation that gives rise to UC and other inflammatory bowel diseases, and restore immune homeostasis with minimal systemic exposure and potentially fewer adverse events compared to systemic administration of IL-10. AMT has amassed a broad global IP portfolio surrounding its multiple technology platforms and therapeutic programs.