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Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum`s proprietary product engine, FulcrumSeek™, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Fulcrum has also advanced FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta-thalassemia into Phase 1 clinical development.
Opexa Therapeutics is a Spring, TX-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology`s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic.
GVI Technical Services is a Twinsburg, OH-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Minerva Neurosciences, Inc. (NASDAQ: NERV) is a clinical-stage biopharmaceutical company focused on the development of a portfolio of product candidates to treat central nervous system (CNS) diseases. Our goal is to transform the lives of patients with improved therapeutic options. Leveraging extensive domain expertise, we have identified and acquired or in-licensed a portfolio of development-stage proprietary compounds with what we believe are innovative mechanisms of action. Our executive management team includes globally recognized specialists in central nervous system diseases addressing significant unmet medical needs. Our strategy is based on the following key principles: • Develop differentiated products based on biological and clinical insights related to the unmet needs of patients; • Conduct proof-of-principle trials in geographic areas with well characterized and validated patient populations where we have access to highly trained physicians with experience in conducting CNS-related trials; • Leverage the randomized, double-blind, placebo-controlled data from these trials to advance the clinical development of our products in multiple regulatory jurisdictions; • Selectively explore collaborations with leading pharmaceutical companies to maximize the value of our current product candidate portfolio, particularly in connection with the initiation of pivotal Phase III clinical trials and subsequent regulatory review, approval and commercialization; • Apply our management team`s expertise and current intellectual property portfolio to identify and explore additional indications relating to our current portfolio of compounds and to acquire additional product candidates