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Ultragenyx is a clinical-stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies. Our company is led by an experienced management team in rare disease therapeutics. Recognizing that our primary responsibility is to our patients, we are working with advocacy groups to provide support and outreach to individuals and families affected by these disorders and engage them in the clinical testing process. We are also working with regulatory agencies to design and conduct high quality clinical studies that meet the requirements for approval. We are creating an improved model for successful rare disease drug development that increases efficiency and effectiveness by changing the way the process is organized and conducted. We believe that we can deliver significant value to our patients by building a diverse and high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine. We are looking for highly motivated individuals to join our team in an exciting biotechnology environment. If you are looking for a meaningful position that has the ability to transform the lives of patients and be part of a high performance team focused on the same goal, this is the opportunity you have been waiting for.
Epizyme is a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative personalized therapeutics for patients with genetically defined cancers. We systematically identify the genetic alterations that create cancer causing genes, called oncogenes, select patients in whom the identified genetic alteration is found, and then design small molecule therapeutic product candidates to inhibit the oncogene. The clinical development plan for each of our therapeutic product candidates is directed towards patients with a particular genetically defined cancer. Our approach is part of a broader trend towards personalized therapeutics based on first identifying the underlying cause of a disease affecting specific patient populations, applying rational drug design tools to create a therapeutic to inhibit a molecular target in the identified disease pathway, and using a companion diagnostic to select the right patients for treatment. We have built a proprietary product platform that we use to create small molecule inhibitors of a 96-member class of enzymes known as histone methyltransferases, or HMTs. Genetic alterations can result in changes to the activity of HMTs, making them oncogenic. When Epizyme was founded, we recognized that the HMT class of enzymes might contain many potential oncogenes and presented the opportunity to discover, develop and commercialize multiple personalized therapeutics. We have prioritized 20 of the 96 HMTs as attractive targets for personalized therapeutics based on their oncogenic potential. Our two most advanced therapeutic product programs target the HMTs DOT1L (for the treatment of acute leukemias with genetic alterations of MLL) and EZH2 (for a genetically defined subtype of non-Hodgkin lymphoma and solid tumors including INI1-deficient tumors). We believe that our ongoing Phase 1 adult trial for EPZ-5676, targeting DOT1L, is the first clinical trial of an HMT inhibitor. In May 2014, we initiated a Phase 1b clinical trial for EPZ-5676 in pediatric patients with MLL-r leukemia, which is considered to be the last largely untreatable pediatric acute leukemia. We are also conducting a Phase 1/2 clinical trial of EPZ-6438, which is being developed for the treatment of non-Hodgkin lymphoma and solid tumors including INI1-deficient tumors such as synovial sarcoma and malignant rhabdoid tumors, or MRT. We were founded in 2007 and are led by a management team with extensive experience in the pharmaceutical industry. We have entered into therapeutic collaborations with Celgene, Eisai and GSK that have provided us with approximately $184 million in non-equity funding. As of June 30, 2014, we had $232.1 million in cash, cash equivalents and accounts receivables.
The mission at Ascus Biosciences is better microbes for a better life. Based in San Diego, CA, Ascus is focused on discovering, developing, and commercializing first-in-class endomicrobial products for the animal health and nutrition industries.
Akero Therapeutics is developing best-in-class medicines that restore metabolic balance to treat non-alcoholic steatohepatitis (NASH) and other serious metabolic diseases.
EGEN is a Huntsville, AL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.