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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.

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.

Helix BioPharma Corp. operates as an immune-oncology company in Canada. It is developing L-DOS47 for the treatment of non-small cell lung cancer; and V-DOS47 that targets the vascular epithelial growth factor receptor 2. Helix BioPharma Corp. has collaboration agreements with Moffitt Cancer Center to investigate the pharmacodynamics of L-DOS47 and determine the benefits of combining L-DOS47 with immune checkpoint inhibitors; and with University Hospital Tuebingen to assess the therapeutic response of L-DOS47 in cancer models expressing CEACAM6, with advanced preclinical metabolic imaging. The company is headquartered in Toronto, Canada.

scPharmaceuticals Inc., a pharmaceutical company, engages in the development and commercialization of various pharmaceutical products. Its lead product candidate is FUROSCIX that consists of formulation of furosemide, which is delivered through an on-body infusor for treatment of congestion due to fluid overload in adults with chronic heart failure and kidney disease, as well as consists of subcutaneous loop diuretic that delivers IV equivalent diuresis at home; and FUROSCIX On-Body Infusor, a drug-device combination product consisting of FUROSCIX. The company's product pipeline also includes SCP-111 (furosemide injection), an investigational pH neutral aqueous furosemide formulation that is being developed for subcutaneous administration outside of the hospital setting, including patient self-administration in the home; and SCP-111 Autoinjector, an investigational single-entity, drug-device combination product candidate consisting of a prefilled syringe containing SCP-111, preloaded into a commercially available, fixed single dose, disposable, two step mechanical autoinjector. It has a collaboration agreement with West Pharmaceutical Services, Inc. The company was incorporated in 2013 and is headquartered in Burlington, Massachusetts.