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We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Center for Functional Genomics is a Rensselaer, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare diseases. Our shared purpose to serve patients in need drives us to find answers, change the world, and create a legacy. Alexion`s three highly innovative therapies treat patients with four severe and ultra-rare diseases, and we are committed to developing additional therapies that have the potential to transform patients` lives. Today, Alexion is advancing the most robust rare disease pipeline in the biotech industry, which, in addition to our complement and metabolic clinical programs, includes more than 30 diverse preclinical programs across a range of therapeutic modalities. Alexion has more than 3,000 employees serving patients in 50 countries. Our global headquarters and research operations are based in New Haven, Connecticut and our EMEA headquarters are in Zürich, Switzerland. We also have global supply chain and operations headquarters in Ireland, manufacturing facilities in the United States, and local and regional operations in countries around the world. Alexion was ranked number 5 by Science magazine in its Top Employer Survey and has also been ranked as one of the top companies on the Forbes "World`s Most Innovative Companies" list every year since 2012.
Magenta Therapeutics is a biotechnology company harnessing the power of stem cell science to revolutionize stem cell transplantation for patients with immune and blood based diseases.
Hookipa Pharma Inc. (NASDAQ: HOOK) is a clinical stage biopharmaceutical company developing a new class of immunotherapeutics, targeting infectious diseases and cancers based on its proprietary arenavirus platform that is designed to reprogram the body`s immune system. HOOKIPA`s proprietary arenavirus-based technologies, VaxWave®*, a replication-deficient viral vector, and TheraT®*, a replication-attenuated viral vector, are designed to induce robust antigen specific CD8+ T cells and pathogen-neutralizing antibodies. Both technologies are designed to allow for repeat administration while maintaining an immune response. TheraT® has the potential to induce CD8+ T cell response levels previously not achieved by other published immuno-therapy approaches. HOOKIPA`s “off-the-shelf” viral vectors target dendritic cells in vivo to activate the immune system. HOOKIPA`s VaxWave®-based prophylactic cytomegalovirus vaccine candidate is currently in a Phase 2 clinical trial in patients awaiting kidney transplantation from living cytomegalovirus-positive donors. To expand its infectious disease portfolio, HOOKIPA has entered into a collaboration and licensing agreement with Gilead Sciences, Inc. to jointly research and develop functional cures for HIV and Hepatitis B infections. HOOKIPA is building a proprietary immuno-oncology pipeline by targeting virally mediated cancer antigens, self-antigens and next-generation antigens.