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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.

CymaBay Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing and providing therapies to treat liver and other chronic diseases. Its lead product candidate is seladelpar (MBX-8025), a selective agonist of peroxisome proliferator activated receptor delta for the treatments of primary biliary cholangitis (PBC). The company also develops MBX-2982, a G protein-coupled receptor 119 (GPR119) agonist, in subjects with type 1 diabetes. It has a license agreement with ABW Cyclops SPV LP to support development of seladelpar for the treatment of PBC. The company was formerly known as Metabolex, Inc. CymaBay Therapeutics, Inc. was incorporated in 1988 and is headquartered in Fremont, California.

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.

RedHill Biopharma Ltd., a specialty biopharmaceutical company, primarily focuses on gastrointestinal and infectious diseases. The company develops and commercializes Talicia for the treatment of H. pylori infection in adults; and Aemcolo for the treatment of travelers' diarrhea in adults. Its pipeline consists of five therapeutic candidates, which are in clinical development include opaganib for treating patients hospitalized with SARS-CoV-2 severe COVID-19 pneumonia, advanced unresectable cholangiocarcinoma, prostate cancer, and nuclear radiation protection; RHB-107 (upamostat) for treating outpatients infected with SARS-CoV-2 (COVID-19 disease) and advanced unresectable cholangiocarcinoma; RHB-104 for Crohn's disease; RHB-102 (Bekinda) for the treatment of acute gastroenteritis and gastritis, irritable bowel syndrome with diarrhea, and oncology support anti-emetic; and RHB-204 for pulmonary nontuberculous mycobacteria infections caused by mycobacterium avium complex. The company was incorporated in 2009 and is headquartered in Tel Aviv, Israel.

Selecta Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the research and development of nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. The company's lead therapeutic gene therapy program is SEL-302 that is in Phase I clinical trial to enhance the treatment of methylmalonic acidemia. It is also developing biologic therapies, such as SEL-212 that is in Phase III clinical trials for the treatment of chronic refractory gout; and product candidates to treat IgA-mediated diseases, including IgA nephropathy, linear IgA bullous dermatitis, IgA pemphigus, and Henoch-Schonlein purpura. In addition, the company is developing gene therapies comprising for the treatment of pompe disease, duchenne muscular dystrophy, limb-girdle muscular dystrophy, lysosomal storage disorder, and other autoimmune diseases. Further, it develops tolerogenic therapies to treat autoimmune diseases. The company has license and collaboration agreements with Ginkgo Bioworks Holdings, Inc.; Genovis AB (publ.); Cyrus Biotechnology, Inc.; IGAN Biosciences, Inc.; Astellas Therapeutics, Inc.; Takeda Pharmaceuticals USA, Inc.; Swedish Orphan Biovitrum AB (publ.); Sarepta Therapeutics, Inc.; Asklepios Biopharmaceutical, Inc.; Massachusetts Institute of Technology; and Shenyang Sunshine Pharmaceutical Co., Ltd. Selecta Biosciences, Inc. was incorporated in 2007 and is headquartered in Watertown, Massachusetts.