ALX Oncology
NASDAQ:ALXOALX Oncology Holdings Inc., a clinical-stage immuno-oncology company, focuses on developing therapies for cancer patients in the United States. The company's lead product candidate is Evorpacept, a CD47 blocking therapeutic biologic in development as a combination therapy with other anti-cancer agents, including ASPEN-06, under Phase 2 clinical study for treating Gastric/GEJ cancer; ASPEN-07, under Phase 1 clinical study for treating urothelial cancer; and ASPEN-03 and ASPEN-04, both under Phase 2 clinical study for treating head and neck squamous cell carcinoma. It also has collaboration agreement for Evorpacept combination programs comprising Jazz Pharmaceuticals plc for zanidatamab, under Phase 1 trial for the treatment of breast cancer and other solid tumors; Quantum Leap Healthcare collaborative with an ADC, fam-trastuzumab deruxtecan-nxki, under Phase 1 trial for the treatment of patients with breast cancer; MD Anderson Cancer Center with rituximab and lenalidomide for the treatment of patients with indolent and aggressive NHL; Sanofi with isatuximab and dexamethasone, under Phase 1/2 trial for the treatment of patients with relapsed or refractory multiple myeloma; Academic Gastrointestinal Cancer Consortium with pembrolizumab and cetuximab, under Phase 2 trial to treat refractory microsatellite stable metastatic colorectal cancer; and University of Pittsburgh with liposomal doxorubicin and pembrolizumab, under Phase 2 trial recurrent platinum-resistant ovarian cancer. In addition, the company has collaboration agreement with Tallac Therapeutics, Inc. for the development of ALTA-002, a potent immune activator targeted to myeloid cells in the tumor to promote innate and adaptive anti-cancer immune responses. It also has license agreements with Board of Trustees of the Leland Stanford Junior University, Selexis SA, and Crystal Bioscience, Inc. The company was incorporated in 2015 and is headquartered in South San Francisco, California.
Generation Bio
NASDAQ:GBIOGeneration Bio Co. develops non-viral genetic medicines for the treatment of rare and prevalent diseases. The company develops cell-targeted lipid nanoparticle (ctLNP) platform, a modular delivery system for nucleic acids to avoid off-target clearance by the liver and spleen that enables ctLNPs to persist in systemic circulation, which allows for highly selective and potent ligand-driven targeting to specific tissues and cell types; and novel immune-quiet DNA (iqDNA) to enable long-lasting high levels of gene expression from non-integrating episomes and avoids innate immune sensors that have long prevented DNA from use in non-viral systems. It uses its platform for developing a portfolio of programs for treating cancer, autoimmune, hematologic disorders, prioritizing sickle cell, beta-thalassemia, and hemophilia A diseases, as well as for other tissues and cell types, including retina, skeletal muscle, and central nervous system. The company was formerly known as Torus Therapeutics, Inc. and changed its name to Generation Bio Co. in November 2017. Generation Bio Co. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.
Intercept Pharmaceuticals
NASDAQ:ICPTIntercept Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics to treat progressive non-viral liver diseases in the United States, Europe, and Canada. The company markets Ocaliva, a farnesoid X receptor agonist used for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults. It is also developing Ocaliva for various indications, including nonalcoholic steatohepatitis and NASH; and other product candidates in various stages of clinical and preclinical development. The company has a license agreement with Aralez Pharmaceuticals Canada Inc. to develop and commercialize bezafibrate in the United States. It markets its products through an internal commercial organization and third-party distributors. The company was incorporated in 2002 and is headquartered in Morristown, New Jersey. As of November 8, 2023, Intercept Pharmaceuticals, Inc. operates as a subsidiary of Alfasigma S.p.A..
Indivior
OTCMKTS:INVVYIndivior PLC is a holding company, which engages in the development, manufacture and sale of buprenorphine-based prescription drugs for treatment of opioid dependence. Its products include Suboxone Film, Suboxone Tablet, and Subutex Tablet. It operates through the following geographical segments: United States, Rest of World, and United Kingdom. The company was founded on September 26, 2014 and is headquartered in Slough, the United Kingdom.
Rocket Pharmaceuticals
NASDAQ:RCKTRocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.