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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.

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.

Northwest Biotherapeutics, Inc., a biotechnology company, develops personalized immune therapies for cancer in the United States and internationally. The company develops its products based on DCVax, a platform technology that uses activated dendritic cells to mobilize a patient's own immune system to attack cancer. Its lead product, DCVax-L, which has completed Phase III clinical trials to treat Glioblastoma multiforme brain cancer. The company also develops DCVax-Direct, which is in Phase I/II clinical trials to treat inoperable solid tumors. The company was founded in 1996 and is headquartered in Bethesda, Maryland.

vTv Therapeutics Inc., a clinical stage biopharmaceutical company, focuses on the development of orally administered treatments for metabolic and inflammatory diseases. The company's lead drug candidate is TTP399, an orally administered, small molecule, and liver-selective glucokinase activator for the treatment of type 1 diabetes that is in Phase III clinical trial; and HPP737, an orally administered non-CNS penetrant phosphodiesterase type 4 (PDE4) inhibitor that addresses inflammatory diseases, psoriasis, COPD, and Atopic Dermatitis that is in Phase III clinical trial. It also develops TTP273, an orally available, small molecule glucagon-like peptide 1 receptor agonists for the treatment of cystic fibrosis related diabetes that is in Phase I clinical trial. In addition, the company is involved in the clinical development of other programs, including HPP3033, a non-electrophilic therapeutic approach to activating the nuclear factor erythroid 2related factor 2 (Nrf2) pathway for the treatment of chronic diseases associated with oxidative stress; and Azeliragon, a RAGE antagonist for inflammatory lung diseases, including severe COVID-19, as well as for glioblastoma, other cancers, and cancer treatment-related conditions. vTv Therapeutics Inc. has a license agreement with Reneo Pharmaceuticals, Inc. to develop and commercialize peroxisome proliferation activated receptor delta agonist program, including the compound HPP593 for therapeutic, prophylactic, and diagnostic application in humans; and with Anteris Bio, Inc. to develop and commercialize HPP971, a Nrf2 activator; with Cantex Pharmaceuticals, Inc.; Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd.; Newsoara Biopharma Co., Ltd.; JDRF International; and Novo Nordisk A/S. The company was incorporated in 2015 and is headquartered in High Point, North Carolina. vTv Therapeutics Inc. is a subsidiary of MacAndrews & Forbes Incorporated.

Zynerba Pharmaceuticals, Inc. operates as a clinical stage specialty pharmaceutical company. The company focuses on developing pharmaceutically produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. It is developing Zygel, a pharmaceutically produced cannabidiol formulated as a permeation-enhanced gel for transdermal delivery. The company was formerly known as AllTranz, Inc. and changed its name to Zynerba Pharmaceuticals, Inc. in August 2014. Zynerba Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Devon, Pennsylvania.