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GenSight Biologics is is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics` pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics` lead product candidate, GS010, is in Phase III trials in Leber`s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics` product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Provista Life Sciences is a Phoenix, AZ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Zygote is a Lindon, UT-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Equillium is a clinical-stage biotechnology company leveraging deep understanding of immunobiology to pioneer new products with a mission to dramatically improve the lives of patients with severe autoimmune and inflammatory diseases who continue to suffer with high unmet medical needs.