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Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company, focuses on developing and delivering gene therapy products for severe and life-threatening rare diseases. The company's lead programs are EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB); ABO-102, which are AAV based gene therapies for Sanfilippo syndrome type A; and ABO-101, an adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome type B. It is also developing ABO-201 gene therapy for juvenile Batten disease; ABO-202 gene therapy for treatment of infantile Batten disease; EB-201 for for epidermolysis bullosa (EB); ABO-301 for Fanconi anemia disorder; and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. Further, it is involved in marketing MuGard, a mucoadhesive oral wound rinse for the management of mucositis, stomatitis, aphthous ulcers, and traumatic ulcers. Abeona Therapeutics Inc. has collaborations with EB Research Partnership and Epidermolysis Bullosa Medical Research Foundation that focus on gene therapy treatments for EB; and Brammer Bio for commercial translation of ABO-102. The company was formerly known as PlasmaTech Biopharmaceuticals, Inc. and changed its name to Abeona Therapeutics Inc. in June 2015. Abeona Therapeutics Inc. was incorporated in 1989 and is based in Dallas, Texas.

Aptinyx Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of novel, proprietary, and synthetic small molecules for the treatment of brain and nervous system disorders. Its lead product candidate NYX-783, an N-methyl-D-aspartate receptors (NMDAr) modulator that is in Phase I clinical trial to treat opioid use disorder, or OUD; and in preclinical trial for the treatment of alcohol use disorder, or AUD. The company has a research collaboration agreement with Allergan plc. Aptinyx Inc. was incorporated in 2015 and is headquartered in Evanston, Illinois.

Corvus Pharmaceuticals, Inc., a clinical stage biopharmaceutical company, focuses on the development and commercialization of immune modulator product candidates to treat solid cancers, T cell lymphomas, autoimmune, allergic, and infectious diseases. Its lead product candidate is soquelitinib (CPI-818), a selective covalent inhibitor of interleukin 2 inducible T cell kinase (ITK), which is in a multi-center Phase 1/1b clinical trial for the treatment of peripheral T cell lymphoma, solid tumors, and atopic dermatitis. The company is also developing ciforadenant (CPI-444), an oral small molecule antagonist of the A2A receptor that is in Phase 2 clinical trial for the treatment of metastatic renal cell cancer; and mupadolimab (CPI-006), a humanized monoclonal antibody, which is in Phase 1b clinical trial for the treatment of non-small cell lung cancer and head and neck cancer. In addition, it is developing CPI-182, an antibody designed to block inflammation and myeloid suppression that is in investigational new drug application-enabling studies, as well as CPI-935, an adenosine A2B receptor antagonist to prevent fibrosis. Corvus Pharmaceuticals, Inc. has a license afreemnt with Monash University to research, develop, and commercialize certain antibodies directed to CXCR2 for the treatment of human diseases; and Vernalis (R&D) Limited to develop, manufacture, and commercialize products containing certain adenosine receptor antagonists, including ciforadenant, as well as strategic collaboration with Angel Pharmaceuticals Co. Ltd. for the development and commercialization of mupadolimab. Corvus Pharmaceuticals, Inc. was incorporated in 2014 and is based in Burlingame, California.

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.