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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.

GlycoMimetics, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of glycobiology-based therapies for cancers, including acute myeloid leukemia (AML) and inflammatory diseases with unmet needs in the United States. It is developing uproleselan, an E-selectin antagonist, which is used in combination with chemotherapy to treat AML, as well as in phase 3 trial to treat relapsed/refractory AML. The company also develops various other programs, including GMI-1687, an antagonist of E-selectin to treat vaso-occlusive crisis; and galectin-3 antagonists, a carbohydrate-binding protein. In addition, it is developing GMI-1359, which targets e-selectin and a chemokine receptor for the treatment of cancers that affect the bone and bone marrow, including solid tumors. The company has a cooperative research and development agreement with the National Cancer Institute; and a collaboration and license agreement with Apollomics (Hong Kong) Limited for the development and commercialization of uproleselan and GMI-1687. GlycoMimetics, Inc. was incorporated in 2003 and is headquartered in Rockville, Maryland.

Neptune Wellness Solutions Inc., a health and wellness products company, specializes in the extraction, purification and formulation of cannabis products, as well as other specialty ingredients, such as MaxSimil, a patented ingredient that enhances the absorption of lipid-based nutraceuticals, and various other marine and seed oils. Its custom formulations are available in various delivery forms, such as softgels, liquid solutions, nutritional emulsions, and chewables. Neptune Wellness Solutions Inc. was founded in 1998 and is headquartered in Laval, Canada.

Ovid Therapeutics Inc., a biopharmaceutical company, engages in the development of impactful medicines for patients and families with epilepsies and seizure-related neurological disorders in the United States. The company is developing soticlestat, a novel cholesterol 24 hydroxylase inhibitor, which is in Phase 3 clinical trials for the potential treatment of patients with resistant epilepsies; OV329, a GABA aminotransferase inhibitor which is in Phase 1 clinical trials for the treatment of seizures associated with tuberous sclerosis complex and infantile spasms; and OV350, a small molecule direct activator of the KCC2 transporter, which is in Phase 1 clinical trials for treating epilepsies. It also develops OV815, that focuses on the mutations associated with KIF1A-associated neurological disorder (KAND); OV888 (GV101), a highly selective rock2 inhibitor which is in Phase 1 double-blind multiple-ascending dose trial; OV825, has advanced to potential candidate lead identification for the rare neurodevelopmental condition HNRNPH2 (Bain Syndrome); and OV882, a short hairpin RNA gene therapy for the treatment of Angelman syndrome. The company has license and collaboration agreements with Healx, AstraZeneca AB, H. Lundbeck A/S, Northwestern University, and Graviton, as well as Marinus Pharmaceuticals, Inc. The company was incorporated in 2014 and is headquartered in New York, New York.

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.