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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.
Allogene Therapeutics is a biotechnology company with a mission to catalyze the next revolution in cancer treatment through the development of allogeneic chimeric antigen receptor T-cell (CAR T) therapy directed at blood cancers and solid tumors. Founded and led by former Kite Pharma executives who bring unrivaled clinical development acumen in cell therapy, Allogene is well-positioned to further the potential of allogeneic cell therapy for patients. Allogeneic CAR T therapies are engineered from cells of healthy donors and stored for “off-the-shelf” use in patients. This approach eliminates the need to create personalized therapy from a patient`s own cells, simplifies manufacturing, and reduces the time patients must wait for CAR T treatment. The Allogene portfolio includes 16 pre-clinical T cell therapy assets and UCART19, an allogeneic CAR T therapy currently in Phase 1 development for the treatment of acute lymphoblastic leukemia (ALL). Through its notable partnerships, Allogene leverages pioneering technology platforms, including TALEN® gene editing technology, to progress its portfolio of immuno-oncology therapies. Allogene, with headquarters in San Francisco, California, is a Two River portfolio company formed with one of the largest Series A financings in biotechnology from an investment consortium that includes TPG, Vida Ventures, BellCo Capital, the University of California Office of the Chief Investment Officer and Pfizer, among others.
Sheffield Bio-Science is a Norwich, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Modis Therapeutics is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with high unmet medical need.
Enzyvant is also advancing the development of RVT-802, an investigational tissue-based biologic therapy for the potential treatment of primary immune deficiency associated with complete DiGeorge Syndrome. RVT-802 has been granted orphan drug designation, Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and pediatric rare disease designation by the U.S. Food and Drug Administration. Enzyvant anticipates a potential BLA filing for RVT-802 in the first half of 2018. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.