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BlackThorn Therapeutics is a neurobehavioral health company developing targeted therapeutics, informed by a deep understanding of brain and behavior relationships, to significantly improve patient outcomes.
CRISPR Therapeutics is a leading gene-editing company focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. Our multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Our lead programs in beta-thalassemia and sickle cell disease have advanced to IND/CTA-enabling studies with a CTA filing planned by the end of 2017, and we are advancing additional programs in ex vivo and in vivo disease areas. In addition to our fully-owned programs, our strategic collaborations with Bayer AG and Vertex Pharmaceuticals expand our portfolio and enable us with unique capabilities. Through our private financings, partnerships, and IPO we have raised >$400M to fund and accelerate our portfolio. We have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from our scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. Our company is headquartered in Zug, Switzerland with R&D operations in Cambridge, Massachusetts, USA and some business operations in London, United Kingdom.
Lysogene is a biopharmaceutical gene therapy company with lead programs in neurodegenerative lysosomal storage disorders that affect children. Founded in 2009 by Karen Aiach, whose own child was affected by a neurodegenerative disease, Lysogene is rooted in a deep and compassionate understanding of the impact these diseases have on patients and families. Karen`s personal experience fuels her determination to deliver real solutions that will improve patient outcomes and enhance quality of life for both patients and caregivers. Over the past 10 years, Lysogene has acquired deep experience in developing gene therapies—from early discovery through the clinical, registration and review phases. Our strong science and execution track record has been validated by our partnership with Sarepta Therapeutics and the support of top-tier investors, with nearly $100m raised since inception.
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.
Vaxxas is a privately held biotechnology company focused on enhancing the performance of existing and next-generation vaccines with its proprietary high-density microarray patch (HD-MAP). Vaxxas is targeting initial applications in infectious disease and oncology. In addition to HD-MAP HexaPro clinical studies of a COVID-19 vaccine patch, Vaxxas is performing demonstration work in preparation for clinical evaluation under contract with the United States Biomedical Advanced Research and Development Authority (BARDA) on pandemic vaccination solutions. Vaxxas core technology was initially developed at the University of Queensland. The company was founded with the completion of an initial equity financing led by OneVentures Innovation Fund I with co-investors Brandon Capital Partners, Brandon BioCatalyst, and US-based HealthCare Ventures, followed by a further financing led by OneVentures. OneVentures Innovation Fund I and Brandon BioCatalyst are supported by the Australian Government’s Innovation Investment Fund (IIF) program. The IIF is an Australian Government venture capital initiative that provides investment capital and managerial expertise through licensed venture capital fund managers to investee companies.