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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.

KALA BIO, Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapies for the treatment of eye diseases. The company's product candidates include KPI-012, which is in Phase 2b clinical trial for the treatment of persistent corneal epithelial defects; EYSUVIS for the short-term treatment of the signs and symptoms of dry eye disease; and INVELTYS, a topical twice-a-day ocular steroid for the treatment of post-operative inflammation and pain following ocular surgery. Its preclinical development product, including KPI-014 for the treatment of rare inherited retinal diseases. The company was formerly known as Kala Pharmaceuticals, Inc. and changed its name to KALA BIO, Inc. in August 2023. KALA BIO, Inc. was incorporated in 2009 and is headquartered in Arlington, Massachusetts.

Spruce Biosciences, Inc., a biopharmaceutical company, focuses on developing and commercializing novel therapies for rare endocrine disorders. The company engages in developing tildacerfont, a non-steroidal therapy to enhance disease control and reduce steroid burden for patients suffering from congenital adrenal hyperplasia (CAH), which is in Phase 2b clinical trial; and to evaluate glucocorticoid reduction in adult patients with classic CAH that is Phase 2b clinical trial. It is also developing tildacerfont for the treatment of pediatric classic congenital adrenal hyperplasia in children that is in Phase 2 clinical trial; and for females with polycystic ovary syndrome, which is in Phase 2 clinical trial. Spruce Biosciences, Inc. has a license agreement with Eli Lilly and Company to research, develop, and commercialize compounds for various pharmaceutical uses; and collaboration and license agreement with Kaken Pharmaceutical Co. Ltd. to develop, manufacture, and commercialize tildacerfont for the treatment of CAH in Japan. The company was incorporated in 2014 and is headquartered in South San Francisco, California.

Tonix Pharmaceuticals Holding Corp., a biopharmaceutical company, focuses on developing, discovering, commercializing, and licensing therapeutics to treat and prevent human disease and alleviate suffering. It markets Zembrace SymTouch and Tosymra for the treatment of acute migraine with or without aura in adults. Its development portfolio focuses on central nervous system disorders. The company's priority is to submit a New Drug Application (NDA) to the FDA for TNX-102 SL (cyclobenzaprine HCl sublingual tablet), which has completed two positive Phase 3 studies for the management of fibromyalgia. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Its TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted breakthrough therapy designation by the FDA. The company's rare disease development portfolio comprises TNX-2900, an intranasal potentiated oxytocin for the treatment of Prader-Willi syndrome (PWS). Its immunology development portfolio includes biologics to address organ transplant rejection, autoimmunity, and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. The company's infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800, in development as a vaccine to protect against COVID-19. Its infectious disease development portfolio also comprises TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals. The company was founded in 2007 and is headquartered in Chatham, New Jersey.