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.
Aptinyx
NASDAQ:APTXAptinyx Inc., a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of novel, proprietary, and synthetic small molecules for the treatment of brain and nervous system disorders. Its lead product candidate NYX-783, an N-methyl-D-aspartate receptors (NMDAr) modulator that is in Phase I clinical trial to treat opioid use disorder, or OUD; and in preclinical trial for the treatment of alcohol use disorder, or AUD. The company has a research collaboration agreement with Allergan plc. Aptinyx Inc. was incorporated in 2015 and is headquartered in Evanston, Illinois.
Five Prime Therapeutics
NASDAQ:FPRXFive Prime Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the discovery and development of innovative protein therapeutics. The company's product candidates comprise Bemarituzumab, an antibody that inhibits fibroblast growth factor receptor 2b, or FGFR2b, which is in Phase 3 clinical trials to treat patients with gastric or gastroesophageal junction and GEJ cancer; and FPA150, a CD8 T cell checkpoint inhibitor antibody that is in Phase 1a/1b clinical trial that targets B7-H4 in various cancers, as well as FPT155, a soluble CD80 fusion protein, which is in Phase 1a/1b clinical trial that enhances co-stimulation of T cells through CD28. Its product candidates also include Cabiralizumab, an antibody that inhibits colony stimulating factor-1 receptor that is in Phase Ia/Ib clinical trials for the treatment of various cancers in combination with Opdivo. The company's BMS-986258, an anti-T cell immunoglobulin and mucin domain-3 antibody, which is in Phase 1/2 clinical trial as a single agent and in combination with Opdivo in patients with advanced malignant tumors. It has license and collaboration agreements with Bristol-Myers Squibb Company, GlaxoSmithKline LLC, INBRX 110 LP, UCB Pharma S.A., and Zai Lab (Shanghai) Co., Ltd.; and license agreements with Galaxy Biotech, LLC, BioWa, Inc. and Lonza Sales AG. The company was founded in 2001 and is headquartered in South San Francisco, 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.
KalVista Pharmaceuticals
NASDAQ:KALVKalVista Pharmaceuticals, Inc., a clinical stage pharmaceutical company, engages in the discovery, development, and commercialization of small molecule protease inhibitors for diseases with unmet needs in the United States. The company's product portfolio comprises small molecule plasma kallikrein inhibitors targeting hereditary angioedema (HAE) and diabetic macular edema (DME); and oral plasma kallikrein inhibitors. Its products include sebetralstat, which is initiation of the Phase 3 KONFIDENT trial as a potential oral, on-demand therapy for HAE attacks; and Factor XIIa, an oral inhibitor program which is in preclinical stage targets an enzyme in HAE. The company is headquartered in Cambridge, Massachusetts.