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Design Therapeutics, Inc. a biopharmaceutical company, researches, designs, develops, and commercializes small molecule therapeutic drugs for the treatment of genetic diseases in the United States. The company utilizes its GeneTAC platform to design and develop therapeutic candidates for inherited diseases caused by nucleotide repeat expansion. Its lead product candidates for potentially disease-modifying treatment comprises Friedreich Ataxia, a monogenic, autosomal recessive, progressive multi-system disease that affects organ systems dependent on mitochondrial function that brings to neurological, cardiac, and metabolic dysfunction; Myotonic Dystrophy Type-1, a dominantly-inherited, monogenic progressive neuromuscular disease affecting skeletal muscle, heart, brain, and other organs; Fuchs Endothelial Corneal Dystrophy, a genetic eye disease characterized by bilateral degeneration of corneal endothelial cells and progressive loss of vision; and Huntington's Disease, a dominantly inherited, monogenic neurodegenerative disease characterized by movement, cognitive, and psychiatric disorders. Design Therapeutics, Inc. was incorporated in 2017 and is headquartered in Carlsbad, California.

Gossamer 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.

PMV Pharmaceuticals, Inc., a precision oncology company, engages in the discovery and development of small molecule and tumor-agnostic therapies for p53 mutations in cancer. It's lead product candidate is PC14586, a small molecule that corrects mutant p53 protein containing the Y220C mutation and restores wild-type p53 function. The company was formerly known as PJ Pharmaceuticals, Inc. and changed its name to PMV Pharmaceuticals, Inc. in July 2013. PMV Pharmaceuticals, Inc. was incorporated in 2013 and is based in Princeton, New Jersey.

PureTech Health plc, a clinical stage biotherapeutics company, engages in the discovery, development, and commercialization of biotechnology and pharmaceutical solutions in the United States. The company is developing LYT-100, currently under Phase 3 stage, to treat idiopathic pulmonary fibrosis (IPF); and LYT-200, a IgG4 monoclonal antibody, currently under Phase 1/2 stage, to inhibit the activity of galectin-9 for treating solid tumors and hematological malignancies. It is also developing LYT-300, an oral allopregnanolone, currently completed Phase 1 stage, to treat depression, anxiety, and related indications; and LYT-310, an oral cannabidiol, currently under preclinical stage, to treat epilepsies and other neurological indications. In addition, the company is developing LYT-503/IMB-150, a non-opioid inflammation targeted disease immunomodulation for the potential treatment of interstitial cystitis, or bladder pain syndrome; and Glyph technology platform, a lymphatic-targeting chemistry platform, which leverages the body's natural lipid absorption and transports the process to orally administer drugs via the lymphatic system. The company was incorporated in 2015 and is headquartered in Boston, Massachusetts.

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease. In addition, it is developing AMT-162, which is in preclinical trial for the treatment of superoxide dismutase 1- amyotrophic lateral sclerosis; AMT-260 that is in preclinical trial to treat temporal lobe epilepsy; AMT-191, which is in preclinical trial for the treatment of fabry disease; AMT-161 that is in preclinical trial to treat amyotrophic lateral sclerosis caused by mutations; AMT-240, which is in preclinical trial to for the treatment of autosomal dominant Alzheimer's disease; and AMT-210 that is in preclinical trial to treat Parkinson's disease. The company was founded in 1998 and is headquartered in Amsterdam, the Netherlands.