Design Therapeutics
NASDAQ:DSGNDesign 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.
Merus
NASDAQ:MRUSMerus N.V., a clinical-stage immuno-oncology company, engages in the development of antibody therapeutics in the Netherlands. Its bispecific antibody candidate pipeline includes Zenocutuzumab (MCLA-128), which is in a phase 2 clinical trials for the treatment of patients with metastatic breast cancer and castration-resistant prostate cancer, as well as in Phase 1/2 clinical trials for the treatment of solid tumors that harbor Neuregulin 1. The company is also developing MCLA-158, which is in a phase I clinical trial for the treatment of solid tumors; MCLA-145, which is in phase 1 clinical trials for the treatment of solid tumors; MCLA-129, which is in phase 1/2 clinical trials for the treatment of patients with advanced non-small cell lung cancer and other solid tumors; and ONO-4685 that is Phase 1 clinical trial to treat relapsed/refractory T cell lymphoma. In addition, it has collaboration agreement with Betta Pharmaceuticals Co. Ltd for the research and development of stage bispecific antibody candidates include MCLA-129; collaboration with Incyte Corporation for the development of MCLA-145; and collaboration with Gilead Sciences, Inc. to discover novel antibody-based trispecific T-cell engagers. The company was incorporated in 2003 and is headquartered in Utrecht, the Netherlands.
NGM Biopharmaceuticals
NASDAQ:NGMNGM Biopharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of novel therapeutics to treat liver and metabolic diseases, retinal diseases, and cancer. The company develops NGM707, an immunoglobulin-like transcript 2/ immunoglobulin-like transcript 4 dual antagonist monoclonal antibody that is in Phase I/II clinical trials for the treatment of patients with advanced metastatic solid tumors; and Aldafermin, an engineered analog of human hormone fibroblast growth factor 19, which is in Phase IIb clinical trials for the treatment of primary sclerosing cholangitis and non-alcoholic steatohepatitis. It also develops NGM120, an GFRAL antagonist antibody, which is in Phase II clinical trial to treat hyperemesis gravidarum; NGM831 and NGM438 which is in phase I/II clinical trial for the treatment of advanced solid tumors; and NGM621, an monoclonal antibody in Phase II clinical trials for the treatment of geographic atrophy. The company has research collaboration, product development, and license agreements with Merck Sharp & Dohme Corp.; and a collaboration agreement with Merck to focus primarily on the identification, research and development of collaboration compounds directed to targets in the fields of ophthalmology and cardiovascular or metabolic, or CVM, disease, including heart failure. NGM Biopharmaceuticals, Inc. was incorporated in 2007 and is headquartered in South San Francisco, California.
uniQure
NASDAQ:QUREuniQure 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.