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Acer Therapeutics is a biotech company, headquartered in Cambridge, MA, that develops repurposed and reformulated medicines for the treatment of ultra-orphan diseases with significant unmet medical needs. Acer`s lead candidate, EDVISO™ (celiprolol), is the first pharmaceutical therapy seeking approval for patients with vascular-type Ehlers-Danlos syndrome (vEDS). Ehlers-Danlos Syndrome is a group of hereditary disorders of connective tissue with no ethnic predisposition. vEDS is a subtype characterized by severe arterial dissections, ruptures and early death. Complications are rare in childhood, but affect 25% of patients before the age of 20, and 80% by the age of 40. Median age of death is estimated to be around 50 years. Patients are diagnosed by clinical symptoms and confirmed by presence of mutations in the COL3A1 gene. There are approximately 2,000 documented patients, but prevalence could be as high as 5,000 patients in the U.S. (Pepin 2014). There are no specific pharmacological treatments for vEDS and medical intervention centers on symptomatic treatment, prophylactic measures and genetic counseling. Acer was awarded orphan drug designation by the FDA in January 2015. Acer`s second candidate, ACER-001, is the first pharmaceutical therapy being developed for patients with Maple Syrup Urine Disease (MSUD). MSUD is a devastating genetic disease – an inborn error of amino acid metabolism. Approximately 800 patients suffer from MSUD in the U.S. and 3,000 worldwide. There are no therapeutic options for MSUD and diet is not enough. Despite careful dietary management, the majority of MSUD patients have chronic neurological and social impairment, as well as life-threatening episodes of intoxication. Acer was awarded orphan drug designation by the FDA in August 2014.
Today`s Merck is a global healthcare leader working to help the world be well. Merck is known as MSD outside of the United States and Canada. Through our prescription medicines, vaccines, biologic therapies, and consumer care and animal health products, we work with customers and operate in more than 140 countries to deliver innovative health solutions. We also demonstrate our commitment to increasing access to healthcare through far-reaching policies, programs and partnerships.
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Cellular Dynamics International, Inc. develops and manufactures human cells in the United States of America, Europe, Japan, and internationally. The company’s proprietary iCell Operating System comprises human cells in multiple cell types (iCell products), human induced pluripotent stem cells (iPSCs), and custom iPSCs and iCell products (MyCell products). It offers iCell Cardiomyocytes, which are purified human heart cells that beat in a dish and behave just like the heart cells found in the human body for in vitro drug discovery, toxicity testing, and chemical safety, as well as in vivo cellular therapeutics research and development, and stem cell banking; and iCell Neurons that are purified human neurons typical of the forebrain for pre-clinical drug discovery, neurotoxicity testing, and disease modeling for Alzheimer’s diseases, Parkinson’s diseases, Huntington’s diseases, and others. The company also provides iCell Endothelial cells, which are interior surface blood vessel cells for use in vascular biology research, such as angiogenesis, atherosclerosis, inflammation, and other life science research; and iCell Hepatocytes that are human liver cells for pre-clinical drug discovery, hepatotoxicity testing, disease modeling, and other life science research. In addition, it offers MyCell products for making iPSCs for stem cell banking, cell modeling of specific ethnic or disease populations, and genetic engineering for in vitro research; and media and reprogramming kits for making iPSCs for limited research use. The company serves biopharmaceutical companies, government research institutions, academic and nonprofit research institutions, contract research organizations, and stem cell banks. Cellular Dynamics International, Inc. was founded in 2004 and is headquartered in Madison, Wisconsin.