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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.

Chinook Therapeutics, Inc. is a clinical-stage biopharmaceutical company, which engages in discovering, developing, and commercializing precision medicines for rare, severe chronic kidney diseases. The company was founded in 2019 and is headquartered in Seattle, WA.

Marinus Pharmaceuticals, Inc., a pharmaceutical company, focuses on development and commercialization of therapeutic products for patients suffering from rare genetic epilepsies and other seizure disorders. It offers ZTALMY (ganaxolone), an oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder for adult and pediatric patient populations in acute and chronic care, and in-patient and self-administered settings. The company's ZTALMY product candidate acts at synaptic and extrasynaptic GABAA receptors, a target for its anti-seizure, antidepressant, and anxiolytic potential. It is developing ganaxolone for treating genetic epilepsy disorders, such as PCDH19-related epilepsy, and tuberous sclerosis complex. Marinus Pharmaceuticals, Inc. has license agreement with Purdue Neuroscience Company and CyDex Pharmaceuticals, Inc.; and collaboration agreement with Orion Corporation and Tenacia Biotechnology (Shanghai) Co., Ltd. Marinus Pharmaceuticals, Inc. was incorporated in 2003 and is headquartered in Radnor, Pennsylvania.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

Spero Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on identifying, developing, and commercializing novel treatments for multi-drug resistant (MDR) bacterial infections and rare diseases in the United States. The company's product candidates include tebipenem pivoxil hydrobromide (HBr), an oral carbapenem-class antibiotic to treat complicated urinary tract infections, including pyelonephritis for adults; SPR206, an intravenous-administered antibiotic against MDR Gram-negative pathogens comprising carbapenem-resistant enterobacterales (CRE), acinetobacter baumannii, and pseudomonas aeruginosa, as well as negative bacterial infections in the hospital setting; and SPR720, a novel oral antibiotic agent for the treatment of non-tuberculous mycobacterial pulmonary disease. It has license agreement with Meiji Seika Pharma Co., Ltd. to support the development of tebipenem HBr; Everest Medicines to develop, manufacture, and commercialize SPR206 in Greater China, South Korea, and Southeast Asian countries; and Vertex Pharmaceuticals Incorporated for patents relating to SPR720, as well as SPR719, an active metabolite. Spero Therapeutics, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.