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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.
Primmune Therapeutics is developing an orally administered TLR7 agonist to stimulate innate immunity in cancer immunotherapy. Our goal is to improve the response rate of patients to current and future immunotherapy medicines.
RAPT Therapeutics is a clinical stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases. Utilizing its proprietary discovery and development engine, the company develops highly selective small molecules that are designed to modulate the fundamental immune responses underlying these diseases. RAPT has rapidly discovered and advanced two unique drug candidates each targeting CCR4, including our lead oncology drug candidate, FLX475, now in clinical development and our lead inflammation drug candidate, RPT193, expected to enter the clinic in the second half of 2019. The company is also pursuing other discovery targets including GCN2 and HPK1 for the treatment of cancer.
Pionyr Immuntherapeutics (formerly Precision Immune Inc.) is developing cancer immunotherapies that target the tumor microenvironment to enhance the body`s antitumor immunity. The company is exploiting novel target discovery and antibody generation platform technologies to create the next generation of immuno-oncology therapeutics. The company`s approach, Myeloid Tuning™, is designed to enhance the immune system`s anti-tumor response by altering the cellular infiltrate of the tumor microenvironment with high specificity.
Eyenovia is a clinical stage, biopharmaceutical company transforming the delivery of therapeutics for the treatment of prominent eye diseases, such as glaucoma, dry eyes, allergic eye disease and many others. Eyenovia`s breakthrough piezo-dispersion and microdosing technology enable a portfolio of first-in-class, next-generation, micro-therapeutics for the eye designed to reduce ocular and systemic toxicity and improve the risk-benefit profile of both new and existing therapeutics.