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Kidney diseases are a severe and growing problem worldwide, with a lack of effective treatments often leading to dialysis, transplantation and high costs to health care systems. In the U.S. alone, kidney diseases affect an estimated 37 million people and account for over $100 billion in annual costs.
We are dedicated to discovering novel biological targets and developing drugs that profoundly improve the lives of people born with rare metabolic defects who are unable to sustain basic biochemical processes necessary for life.
OraPharma , Inc. is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.
We are a biotechnology company creating a new class of targeted gene therapies for debilitating and life-threatening genetic disorders in both central nervous system (CNS) and non-CNS indications. Current gene therapy approaches have shown dramatic efficacy in several rare diseases but are hindered by imprecise targeting. The inability to selectively transduce specific cells and tissues effectively drives administration of higher doses and resulting safety liabilities. We are addressing these concerns with our proprietary adeno-associated virus (AAV) engineering platform that generates capsids optimized to target specific tissues and cells in the disease organ while limiting transduction of tissues and cells not relevant to the target disease. Through our next-generation AAV platform combined with cargo development and state-of-the-art manufacturing, we are tackling monogenetic and sporadic CNS and non-CNS disorders that have previously been challenging to address.
EMD Serono is the North America biopharma business of Merck KGaA, Darmstadt, Germany, employing over 1,200 individuals around the country with U.S. headquarters located in Rockland, Massachusetts. The company develops and offers therapies for specialty-care conditions, like multiple sclerosis, infertility and cancer. EMD Serono is committed to transforming lives by developing therapies that patients need and providing industry-leading educational and support programs.