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We are a biopharmaceutical company specializing in the development of novel therapeutics to treat rare metabolic diseases. Our lead product candidate is INZ-701, an enzyme replacement therapy (ERT) in the early stages of clinical development for the potential treatment of patients with a variety of calcification disorders linked primarily to mutations in the ENPP1 and ABCC6 genes, or ENPP1 Deficiency and ABCC6 Deficiency respectively. We have demonstrated proof of concept with INZ-701 in both GACI and ARHR2 two phenotypes of ENPP1 Deficiency.
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.
Legacy Health System is an Oregon-based not-for-profit, tax-exempt corporation that includes five full-service hospitals and a children's hospital.
Heat Biologics is a clinical-stage company focused on developing its proprietary ImPACT™ (Immune Pan-Antigen Cytotoxic Therapy), a fully allogeneic (“off-the-shelf”) cell-based immunotherapy for use in the treatment of a wide range of cancers.
Stealth BioTherapeutics is a privately held biopharmaceutical company committed to bringing mitochondria therapies to patients to treat both common and rare diseases. As a key common element in a variety of serious, debilitating diseases, mitochondria – the cell’s energy source – offer a promising, and yet untapped target to modify diseases with significant unmet treatment needs. Stealth is expanding its clinical development program to additional therapeutic areas, including cardio-renal diseases, ophthalmic disorders and orphan mitochondrial diseases. By defining the broad potential of its mitochondrial platform and therapies, Stealth is leading mitochondrial medicine.