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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.

AMAG Pharmaceuticals, Inc., a pharmaceutical company, develops, manufactures, and commercializes therapeutics for maternal and women's health, and anemia management in the United States. The company markets Feraheme (ferumoxytol), an intravenous iron replacement therapeutic agent for the treatment of iron deficiency anemia in adult patients who have intolerance to oral iron or have had unsatisfactory response to oral iron, as well as patients who have chronic kidney disease; Makena, a hydroxyprogesterone caproate injection to reduce the risk of preterm birth in women pregnant with a single baby who have a history of singleton spontaneous preterm birth; Intrarosa(prasterone) vaginal insert steroid for the treatment of dyspareunia due to menopause; and Vyleesi (bremelanotide), an auto-injector device for the treatment of hypoactive sexual desire disorder in pre-menopausal women. Its product candidates also include AMAG-423 (ovine), an antibody fragment, which is in Phase 2b/3a trial for the treatment of severe preeclampsia in pregnant women; and Ciraparantag, an anticoagulant reversal agent that is in planned Phase 2b trial for the treatment of novel oral anticoagulants or low molecular weight heparin. The company sells Feraheme to authorized wholesalers and specialty distributors. The company has license agreements with Endoceutics, Inc., Palatin Technologies, Inc., Velo Bio, LLC, Prasco, LLC, Perosphere Pharmaceuticals Inc., and Antares Pharma, Inc. AMAG Pharmaceuticals, Inc. was founded in 1981 and is headquartered in Waltham, Massachusetts.

Chiasma, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing oral medications using transient permeability enhancer technology platform for the treatment of rare and serious chronic disease worldwide. The company offers MYCAPSSA, an oral octreotide capsule for the long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide. It also develops MPOWERED, an oral octreotide capsule, which has completed Phase III clinical trials for the maintenance treatment of adult patients with acromegaly. Chiasma, Inc. was incorporated in 2001 and is headquartered in Needham, Massachusetts.

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.