BioMarin Pharmaceutical Inc. is a biotechnology company that specializes in developing and commercializing innovative therapies for rare and life-threatening diseases. Founded in 1996 and headquartered in San Rafael, California, BioMarin's product portfolio includes several approved treatments for conditions such as mucopolysaccharidosis I, mucopolysaccharidosis VI, and phenylketonuria (PKU). Key products include Aldurazyme, Naglazyme, Kuvan, Brineura, and Palynziq. The company has also expanded its offerings with recent approvals such as Voxzogo for achondroplasia and Roctavian, a gene therapy for hemophilia A. In addition to its commercial products, BioMarin has a robust pipeline of clinical and pre-clinical candidates targeting various rare diseases. The company collaborates with other organizations to further advance gene therapies and enhance treatment options for patients. BioMarin distributes its products through specialty pharmacies and directly to hospitals and international agencies across various regions.
Prosensa, also known as Ribpharm Inc, is a biotech company specializing in the discovery, development, and commercialization of RNA-based therapeutics. The company is particularly focused on addressing genetic disorders, with an emphasis on neuromuscular conditions, as well as anti-infectives and oncology. Prosensa aims to leverage its strong intellectual property position to capitalize on emerging market opportunities in the healthcare sector. To optimize its operations, the company adopts a focused business model by outsourcing most activities to specialized organizations. Through its innovative approach, Prosensa seeks to bring novel and commercially viable therapeutic products to market.
Zacharon Pharmaceuticals
Acquisition in 2013
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
Acquisition in 2010
ZyStor Therapeutics, Inc. is a privately-held biotechnology company focused on developing enzyme replacement therapies for lysosomal storage diseases. The company specializes in creating innovative replacement technologies that enhance the delivery of therapeutics designed to substitute for missing lysosomal enzymes. These therapies aim to address the needs of patients suffering from genetic disorders characterized by lysosomal storage dysfunction.
LEAD Therapeutics
Acquisition in 2010
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.
Inozyme
Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.
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