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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.

Cerecor, Inc. is a biopharmaceutical company, which engages in the development and commercialization of treatments for rare and orphan diseases. Its lead product candidates include CERC-801, CERC-802, and CERC-803, which are therapies for inherited metabolic disorders known as congenital disorders of glycosylation. The company was founded by Blake M. Paterson, Isaac Blech, Barbara S. Slusher, and Solomon H. Snyder on January 31, 2011 and is headquartered in Rockville, MD.

Checkpoint Therapeutics, Inc., a clinical-stage immunotherapy and targeted oncology company, focuses on the acquisition, development, and commercialization of novel treatments for patients with solid tumor cancers in the United States and internationally. The company's lead antibody product candidate is Cosibelimab, which is in Phase 1 clinical trial to treat patients with selected recurrent or metastatic cancers; and CK-302, a product candidate in preclinical trials for hematological malignancies and solid tumors. It is also developing Olafertinib, which is in Phase 3 clinical trials for the treatment of adult patients with metastatic non-small cell lung cancer; CK-103 for the treatment of various advanced and metastatic solid tumor cancers; and Anti-Carbonic Anhydrase IX (CAIX) antibody, a product candidate in preclinical trials to recognize CAIX expressing cells and kill them via antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity. The company has licensing and collaboration agreements with Adimab, LLC for the development of Cosibelimab; NeuPharma, Inc., to develop and commercialize Olafertinib; and Jubilant Biosys Limited for the development and commercialization of novel compounds that inhibit BET bromodomains. The company was incorporated in 2014 and is based in Waltham, Massachusetts. Checkpoint Therapeutics, Inc. is a subsidiary of Fortress Biotech, Inc.

Eton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.

Otonomy, Inc. operates as a biopharmaceutical company. engages in the development of therapeutics for neurotology. The firm's product pipeline includes OTIVIDEX (dexamethasone) Meniere's disease, OTIPRIO (ciprofloxacin otic suspension) acute otitis media with tubes (AOMT), OTO-313 (gacyclidine) tinnitus, OTO-413 (BDNF) hidden hearing Loss, OTO-510 (otoprotectant) prevent CIHL, OTO-6XX (hair cell regeneration) severe hearing loss. It operates under the Otiprio brand. The company was founded by Jay B. Lichter, Jeffrey Harris, Rick Friedman, and Allen F. Ryan on May 6, 2008 and is headquartered in San Diego, CA.