Abeona Therapeutics
NASDAQ:ABEOAbeona 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.
Agile Therapeutics
NASDAQ:AGRXAgile Therapeutics, Inc., a women's healthcare company, researches, develops, and commercializes prescription contraceptive products for women in the United States. Its lead product is Twirla, also known as AG200-15, a once-weekly prescription combination hormonal contraceptive patch. The company is also developing a pipeline of Twirla line extensions and other products, including AG200-15 Extended Regimen (ER), a regimen that allows a woman to have four episodes of withdrawal bleeding per year; AG200-15 smaller patch (SmP), which is a regimen designed to provide shorter and lighter withdrawal bleeds, and enhance contraceptive efficacy; AG200-15 ER SmP, a regimen to allow a woman to extend the length of her contraceptive cycle, as well as have shorter and lighter withdrawal bleeding episodes per year; and P-Patch, a progestin-only contraceptive patch intended for use by women who are unable or unwilling to take estrogen. The company was incorporated in 1997 and is headquartered in Princeton, New Jersey.
CymaBay Therapeutics
NASDAQ:CBAYCymaBay 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.
Hookipa Pharma
NASDAQ:HOOKHOOKIPA Pharma Inc., a clinical-stage biopharmaceutical company, develops immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform. The company's lead infectious disease product candidate is HB-101, which is in a randomized double-blinded Phase 2 clinical trial in cytomegalovirus-negative patients awaiting kidney transplantation from cytomegalovirus-positive donors. Its lead oncology product candidates are HB-201 and HB-202 that are in preclinical studies for the treatment of human papillomavirus-positive cancers. The company has a collaboration with Gilead Sciences, Inc. to develop arenavirus based therapeutics to treat hepatitis B virus and human immunodeficiency virus infections. HOOKIPA Pharma Inc. was founded in 2011 and is headquartered in New York, New York.
Selecta Biosciences
NASDAQ:SELBSelecta 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.