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 is a biotech company also known as Ribpharm Inc dedicated to the commercialization of products for the healthcare market based on RNA-based therapeutics. During the development of this technology, it became clear that many opportunities exist for the development of these products into novel commercially attractive products. These products are mainly in the field of genetic disorders, anti-infectives and oncology. Prosensa will focus to maximize the commercial development of the current program, and take advantage of the large novel market opportunities supported by a strong IP position. Prosensa will initially operate mainly as a focussed company, outsourcing most of its activities to specialized organizations.
Zacharon Pharmaceuticals
Acquisition in 2013
Zacharon Pharmaceuticals is leveraging unique glycobiology expertise to develop an entirely new class of human therapeutics targeting glycans. Zacharon was incorporated in 2004 and has received initial funding through NIH grants and individual investors. Series A financing was more recently provided by Avalon Ventures, a long-standing San Diego-based venture capital firm with a focus on early stage life science companies. With this strong financial backing and innovative drug development platform in place, Zacharon is developing an entirely new class of human therapeutics addressing substantial unmet needs.
Zystor
Acquisition in 2010
ZyStor Therapeutics, Inc. is a privately-held biotechnology company developing enzyme replacement therapies for the treatment of lysosomal storage diseases.
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 is a provider of therapies for rare diseases of calcification affecting soft tissues and bone created to offer potentially disease-modifying therapies to help children who are affected with rare, but severe and debilitating disorders of metabolism. The company's enzyme replacement therapy provides the treatment of Generalized Arterial Calcification of Infancy (GACI) and Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2), enabling patients to treat diseases characterized by mineral imbalances in the body.
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