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REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.
Code Bio was founded on the belief that more can be done for people living with debilitating genetic diseases. We are driven by the potential of our transformative technology to treat the untreatable and cure the incurable. Code Bio is developing highly targeted genetic medicines with its proprietary, novel, multivalent, synthetic DNA delivery platform, 3DNA®, which has been engineered to overcome many of the challenges inherent with the delivery of genetic medicines. 3DNA offers unparalleled tissue and cell targeting specificity, improved bioavailability, capability to deliver large genetic payloads, potential ability to re-dose, and a scalable, modular, reproducible manufacturing process. Our 3DNA delivery platform is poised to transform the field of genetic medicines, enabling targeted delivery of gene therapy, RNAi and other genetic modalities. Currently we are advancing an internal pipeline of genetic medicines focused on select diseases without a cure (Duchenne`s Muscular Dystrophy and Type 1 Diabetes), as well as establishing partnerships to take forward programs in both rare and prevalent diseases.
XOMA is a late-stage biotechnology company with a diverse portfolio of innovative therapeutic antibodies. The Company has built an expertise in allosteric modulation and has applied that expertise to expand the therapeutic potential of monoclonal antibodies. The first compound from XOMA’s allosteric modulating antibody program is gevokizumab, an IL-1 beta modulating antibody. XOMA has partnered with SERVIER, a global pharmaceutical company based in France, to develop and commercialize gevokizumab for the global market, and the companies are conducting a global Phase 3 program in people with Behçet’s disease uveitis and non-infectious uveitis. Each company also has a proof-of-concept (POC) clinical program in place to identify other IL-1 mediated diseases that could be treated with gevokizumab. One of these POC studies led XOMA to select its next Phase 3 indication, pyoderma gangrenosum, a rare ulcerative skin disease. XOMA`s scientific research also produced the XMet program, which consists of three classes of preclinical allosteric modulating antibodies, including Selective Insulin Receptor Modulators (SIRMs) that could have a major impact on the treatment of diabetes. XOMA will retain the compound that has potential to treat several rare insulin dysfunction-related diseases and to out-license the compounds that could address the diabetes markets.
resTORbio, Inc. is a clinical stage company, developing medicines to treat aging-related diseases and conditions. resTORbio`s lead program targets the mechanistic target of rapamycin complex 1 (mTORC1) pathway to treat aging-related diseases and conditions. Initially, resTORbio is focused on developing medicines that address conditions caused by immunosenescence, the decline in immune function due to aging. resTORbio has initiated a Phase 2b trial for its lead product candidate, RTB101, evaluating its potential to reduce the incidence of respiratory tract infections (RTI) in elderly individuals at increased risk of RTI-related morbidity and mortality. resTORbio`s lead program is built upon two Phase 2a clinical studies demonstrating promising safety and efficacy results in almost 500 elderly individuals.
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.