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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.

Adamas Pharmaceuticals, Inc. engages in discovery and development of chrono-synchronous therapies for chronic neurologic disorders. Its products include GOCOVRI, ADS-5012, ADS-4101, and Namzaric. GOCOVRI is indicated for the treatment of dyskinesia in patients with Parkinson's disease receiving levodopa-based therapy. ADS-5102 is a treatment for walking impairment in patients with multiple sclerosis. ADS-401 treats partial onset seizures in patients with epilepsy. Namzaric is a drug treatment for moderate to severe Alzheimer's disease. The company was founded by Gregory T. Went on November 15, 2000 and is headquartered in Emeryville, CA.

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.

Strongbridge Biopharma Plc operates as a commercial-stage biopharmaceutical company, which focuses on the development and commercialization of therapies for rare diseases. Its pipeline is comprised of Keveyis, Macrilen, Recorlev, and Veldoreotide. The company was founded in 1996 and is headquartered in Trevose, PA.

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.