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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.
Stoke Therapeutics is pioneering a new way to treat the underlying causes of severe #geneticdiseases by precisely upregulating protein expression. We develop antisense oligonucleotide medicines that increase gene expression to treat genetic epilepsies and other severe monogenic diseases, including genetic conditions affecting the central nervous system, eye, liver and kidney. At Stoke, we believe our proprietary technology platform, Targeted Augmentation of Nuclear Gene Output, or #TANGO, will allow us to deliver disease-modifying therapies in a highly precise, durable and controlled manner. We have identified approximately 2,900 genetic diseases which we believe may be amenable to TANGO. At Stoke, we are united around a common goal: to improve the lives of people living with severe genetic diseases. Stoke is located in Bedford, MA with offices in Cambridge, MA.
Sage Therapeutics is a clinical-stage biopharmaceutical company committed to developing novel medicines to transform the lives of patients with life-altering central nervous system (CNS) disorders. Sage`s lead product candidate, ZULRESSO(TM) (brexanolone) injection, has completed Phase 3 clinical development for postpartum depression and a New Drug Application is currently under review with the U.S. Food and Drug Administration. Sage is developing a portfolio of novel product candidates targeting critical CNS receptor systems, including SAGE-217, which is in Phase 3 development in major depressive disorder and postpartum depression.
Madeira Therapeutics is a Overland Park, KS-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Switch Therapeutics is an emerging preclinical stage biotechnology company pioneering a new type of medicine that integrates nucleic acid nanotechnology and RNA interference (RNAi) science with the goal of treating a range of diseases – affecting the central nervous system and systemic indications – with significant unmet needs. Switch`s novel gene knockdown approach is based on technology developed by renowned researchers in the field of RNA from Caltech, Harvard and City of Hope. Based on the scientific discovery of Switch`s co-founders, the company has developed a novel proprietary platform known as CASi (Conditionally Activated siRNAs) that combines advantageous properties of both single and double-stranded RNAs in a single molecule, allowing for cell selective RNAi activity. To date, Switch has raised $52M in funding through Series A, and the company`s South San Francisco based team has continued to grow as its research has advanced.