BioMarin Pharmaceutical Inc. is a biotechnology company that specializes in developing and commercializing therapies for serious and life-threatening rare diseases. Its product portfolio includes several approved therapies such as Aldurazyme for mucopolysaccharidosis I, Brineura for late infantile neuronal ceroid lipofuscinosis type 2, and Kuvan for phenylketonuria (PKU). Other notable products are Naglazyme for mucopolysaccharidosis VI, Palynziq for adult patients with PKU, and Vimizim for mucopolysaccharidosis IV Type A. BioMarin's pipeline features promising candidates, including valoctocogene roxaparvovec for severe hemophilia A and vosoritide for achondroplasia. The company serves a diverse range of customers, including specialty pharmacies, hospitals, government agencies, and pharmaceutical distributors across the United States, Europe, and Latin America. BioMarin has established collaborations and license agreements with various organizations to enhance its product development efforts. Founded in 1996 and headquartered in San Rafael, California, BioMarin remains committed to advancing therapies for rare genetic conditions.
Prosensa is a biopharmaceutical company that specializes in the discovery, development, and commercialization of RNA-based therapeutics. It is particularly focused on addressing genetic disorders, with a primary emphasis on neuromuscular conditions. The company recognizes significant market opportunities in the healthcare sector for innovative products targeting genetic disorders, anti-infectives, and oncology. To maximize commercial potential, Prosensa plans to leverage its strong intellectual property position while primarily outsourcing its operations to specialized organizations. Through this strategy, the company aims to develop novel therapeutics that can address unmet medical needs in various therapeutic areas.
Zacharon Pharmaceuticals is a biotech company specializing in the development of therapeutics that target glycans, a class of carbohydrates critical to various biological processes. Founded in 2004, the company combines its expertise in glycobiology with a drug discovery platform to create small molecules that influence glycan and glycolipid metabolism. Initial funding came from NIH grants and individual investors, while recent Series A financing was secured from Avalon Ventures, a venture capital firm known for supporting early-stage life sciences companies. With this financial support and a focus on addressing significant unmet medical needs, Zacharon Pharmaceuticals aims to pioneer a new class of human therapeutics.
ZyStor Therapeutics, Inc. is a privately-held biotechnology company developing enzyme replacement therapies for the treatment of lysosomal storage diseases.
LEAD Therapeutics, Inc. is a chemistry-driven drug discovery company focused on delivering novel and differentiated small molecule IND candidates against clinically validated therapeutic targets, focused primarily on the treatment of Cancer and infectious diseases.
Huxley Pharmaceuticals
Acquisition in 2009
Huxley Pharmaceuticals is private life sciences company founded and managed by Aceras BioMedical, LLC. Aceras is dedicated to funding and developing novel innovations that address unmet medical needs.
Glyko Biomedical
Acquisition in 2002
Glyko Biomedical Ltd. offers scientific research services aimed at developing novel analytic research instrumentation.
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