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.
Gossamer Bio
NASDAQ:GOSSGossamer Bio, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing seralutinib for the treatment of pulmonary arterial hypertension (PAH) in the United States. The company is developing GB002, an inhaled, small molecule, platelet-derived growth factor receptor, or PDGFR, colony-stimulatin factor 1 receptor and c-KIT inhibitor, which is in Phase 3 clinical trial for the treatment of PAH. It has license agreements with Pulmokine, Inc. to develop and commercialize GB002 and related backup compounds. The company was formerly known as FSG, Bio, Inc. and changed its name to Gossamer Bio, Inc. in 2017. Gossamer Bio, Inc. was incorporated in 2015 and is headquartered in San Diego, California.
Phathom Pharmaceuticals
NASDAQ:PHATPhathom Pharmaceuticals, Inc., biopharmaceutical company, focuses on developing and commercializing treatments for gastrointestinal diseases. The company has the rights in the United States, Europe, and Canada for an investigational potassium-competitive acid blocker (P-CAB) that blocks acid secretion in the stomach. It is also developing vonoprazan for the treatment of erosive gastroesophageal reflux disease; and in combination with antibiotics for the treatment of Helicobacter pylori infection. The company was incorporated in 2018 and is headquartered in Florham Park, New Jersey.
Pliant Therapeutics
NASDAQ:PLRXPliant Therapeutics, Inc., a clinical stage biopharmaceutical company, discovers, develops, and commercializes novel therapies for the treatment of fibrosis and related diseases in the United States. The company's lead candidate is bexotegrast, an oral, small-molecule, dual selective inhibitor of avß6 and avß1 integrins, which is in phase 2b trials for idiopathic pulmonary fibrosis and in phase 2a trial for primary sclerosing cholangitis. It also develops PLN-1474, an oral, small-molecule selective inhibitor of avß1 for the treatment of liver fibrosis associated with nonalcoholic steatohepatitis; PLN-101095, a dual inhibitor of integrins avß8 and avß1 for the treatment of solid tumors; and PLN-101325 for treatment of muscular dystrophies. Pliant Therapeutics, Inc. was incorporated in 2015 and is based in South San Francisco, California.
Travere Therapeutics
NASDAQ:RTRXTravere Therapeutics, Inc. is a biopharmaceutical company. It engages in the identification, development, commercialization, and distribution of therapies to people living with rare diseases. Its products include Chenodal, Cholbam, and Thiola. The company was founded by Martin Shkreli on February 8, 2008 and is headquartered in San Diego, CA.