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.
Bicycle Therapeutics
NASDAQ:BCYCBicycle Therapeutics plc, a clinical-stage biopharmaceutical company, develops a class of medicines for diseases that are underserved by existing therapeutics in the United States and the United Kingdom. Its product pipeline comprising BT8009, a bicycle toxin conjugate (BTC) which is in phase I/II/III clinical trial for the treatment of high nectin-4 expressing tumors; BT5528, a BTC which is in phase I/II clinical trial for the treatment of Ephrin type A receptor 2 expressing tumor; BT7480, a Bicycle TICA molecule which is in phase I/II targeting Nectin-4 and agonizing CD137; and BT7455, a Bicycle TICA molecule targeting Ephrin type A receptor 2 and CD137 and is in preclinical trial. The company also developing BT1718, a bicycle toxin conjugate (BTC), which is in Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease; and BT7401, a multivalent bicycle CD137 agonist which is in phase IIa to treat advanced solid tumors. In addition, it develops Novel anti-infective, which is in preclinical trial targeting anti-infectives disease; CNS targets, which is in preclinical trial targeting CNS disease; and Novel neuromuscular targets, which is in preclinical trial targeting neuromuscular disease. Further, the company collaborates with biopharmaceutical companies and organizations to develop programs in therapeutic areas. It has collaboration and license agreement with Bayer Consumer Care AG; Novartis Pharma AG; Cancer Research UK; Cancer Research Technology Ltd; and Oxurion NV. Bicycle Therapeutics plc was incorporated in 2009 and is headquartered in Cambridge, the United Kingdom.
IDEAYA Biosciences
NASDAQ:IDYAIDEAYA Biosciences, Inc., a synthetic lethality-focused precision medicine oncology company, discovers and develops targeted therapeutics for patient populations selected using molecular diagnostics in the United States. The company's products in development include IDE196, a protein kinase C inhibitor that is in Phase 2/3 clinical trials for genetically defined cancers having GNAQ or GNA11 gene mutations; IDE397, a methionine adenosyltransferase 2a inhibitor that is in Phase 1/2 clinical trials for patients with solid tumors having methylthioadenosine phosphorylase gene deletions, such as non-small cell lung, bladder, gastric, and esophageal cancers; IDE161, a poly ADP-ribose glycohydrolase inhibitor that is in Phase 1 clinical trial to treat tumors with homologous recombination deficiency (HRD), and other genetic or molecular signatures; GSK101, a Pol Theta Helicase inhibitor that is in Phase 1 clinical trial for the treatment of tumors with BRCA or other homologous recombination, and HRD mutations; and Werner Helicase inhibitors for tumors with high microsatellite instability. It offers preclinical research programs focused on pharmacological inhibition; DECIPHER dual CRISPR library for synthetic lethality target and biomarker discovery; and INQUIRE chemical library and HARMONY machine-learning engines to enhance its drug discovery platform. The company has strategic alliances with GlaxoSmithKline, Pfizer Inc., Novartis International Pharmaceuticals Ltd., Amgen Inc., Gilead Sciences, Inc., Cancer Research UK, and the University of Manchester. IDEAYA Biosciences, Inc. was incorporated in 2015 and is headquartered in South San Francisco, California.
Organigram
NASDAQ:OGIOrganigram Holdings Inc., through its subsidiaries, engages in the production and sale of cannabis and cannabis-derived products in Canada. It offers medical cannabis products, including whole flower, milled flower, pre-rolls, infused pre-rolls, vapes, gummies, and concentrates for medical retailers; adult use recreational cannabis under the SHRED, Holy Mountain, Big Bag O' Buds, Monjour, Trailblazer, SHRED'ems, Edison Cannabis Co., Edison JOLTS, Tremblant, and Laurentian brands. The company also engages in the wholesale shipping of cannabis plant cuttings, dried flowers, blends, pre-rolls, and cannabis derivative-based products to retailers and wholesalers for adult-use recreational cannabis. It sells its products through online, as well as consumer channels. The company was incorporated in 2010 and is headquartered in Toronto, Canada.
ProQR Therapeutics
NASDAQ:PRQRProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.