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Athersys, Inc., a biotechnology company, focuses on the research and development activities in the field of regenerative medicine. Its clinical development programs are focused on treating neurological conditions, cardiovascular diseases, inflammatory and immune disorders, and pulmonary and other conditions. The company's lead platform product includes MultiStem cell therapy, an allogeneic stem cell product for the treatment of patients suffering from neurological damage from an ischemic stroke, as well as for acute respiratory distress syndrome, trauma complications, HSC transplant support, and other indications. It also develops MultiStem cell therapy to promote tissue repair and healing for animal patients. The company has license and collaboration agreements with Healios K.K. to develop and commercialize MultiStem cell therapy for ischemic stroke, acute respiratory distress syndrome, and ophthalmological indications, as well as for the treatment of liver, kidney, pancreas, and intestinal tissue diseases; and the University of Minnesota to develop MultiStem cell therapy platform. The company was founded in 1995 and is headquartered in Cleveland, Ohio.

Fulcrum Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing products for improving the lives of patients with genetically defined diseases in the areas of high unmet medical need in the United States. Its product candidates are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy is under phase III clinical trial; and pociredir, a fetal hemoglobin inducer for the treatment of sickle cell disease and beta-thalassemia is under phase I clinical trial. The company is also discovering drug targets for the treatments of rare neuromuscular, muscular, central nervous system, and hematologic disorders, as well as cardiomyopathies and pulmonary diseases. Fulcrum Therapeutics, Inc. has collaboration and license agreement with Acceleron Pharma Inc. to identify biological targets to modulate specific pathways associated with a targeted indication within the pulmonary disease space; and MyoKardia, Inc. to discover and develop therapies for the treatment of genetic cardiomyopathies. Fulcrum Therapeutics, Inc. was Incorporated in 2015 and is headquartered in Cambridge, Massachusetts.

MEI Pharma, Inc., a clinical-stage pharmaceutical company, focuses on the development and commercialization of various therapies for the treatment of cancer. The company develops Zandelisib, an oral phosphatidylinositol 3-kinase delta inhibitor for the treatment of patients with relapsed/refractory follicular lymphoma; and Voruciclib, an oral cyclin-dependent kinase 9 inhibitor, which is in Phase I clinical trial for acute myeloid leukemia and B-cell malignancies. It also develops ME-344, a mitochondrial inhibitor targeting the oxidative phosphorylation complex which has completed Phase I clinical trial for the treatment of human epidermal growth factor receptor 2 negative breast cancer. MEI Pharma, Inc. has a license agreement with Presage Biosciences, Inc. The company was formerly known as Marshall Edwards, Inc. and changed its name to MEI Pharma, Inc. in July 2012. MEI Pharma, Inc. was incorporated in 2000 and is headquartered in San Diego, California.

Orphazyme A/S, a biopharmaceutical company, develops therapies for the treatment of neurodegenerative orphan diseases. The company focuses on the amplification of heat-shock proteins to develop and commercialize therapeutics for diseases caused by protein misfolding and aggregation, and lysosomal dysfunction, including lysosomal storage and neuromuscular degenerative diseases. Its lead candidate is the Arimoclomol, which is in clinical development for four orphan diseases, including Niemann-Pick disease type C, Amyotrophic Lateral Sclerosis, Inclusion Body Myositis, and Gaucher disease. The company was incorporated in 2009 and is headquartered in Copenhagen, Denmark.

UroGen Pharma Ltd., a biotechnology company, engages in the development and commercialization of solutions for urothelial and specialty cancers. It offers RTGel, a novel proprietary polymeric biocompatible, reverse thermal gelation hydrogel technology to improve therapeutic profiles of existing drugs; and Jelmyto for pyelocalyceal solution. The company's lead product candidate is UGN-102 for the treatment of several forms of non-muscle invasive urothelial cancer that include low-grade upper tract urothelial cancer and low-grade intermediate risk non-muscle invasive bladder cancer (NMIBC). It is also developing UGN-301 for the treatment of high-grade NMIBC. The company has license agreement with Agenus Inc. to develop, make, use, sell, import, and commercialize products of Agenus for the treatment of cancers of the urinary tract via intravesical delivery; strategic research collaboration agreement with MD Anderson focusing on the sequential use of UGN-201 and UGN-301 for the treatment of NMIBC; and licensing and supply agreement with medac Gesellschaft für klinische Spezialpräparate m.b.H. to develop UGN-103 in low-grade intermediate risk NMIBC and UGN-104 in low-grade upper tract urothelial carcinoma. UroGen Pharma Ltd. was incorporated in 2004 and is based in Princeton, New Jersey.