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.
MorphoSys
NASDAQ:MORMorphoSys AG, together with its subsidiaries, engages in the development and commercialization of therapeutics for patients suffering from various cancers in Europe, Asia, and the United States. The company's product pipeline includes Pelabresib that is in Phase 3 trials to treat myelofibrosis and thrombocythemia; Tulmimetostat, a product candidate in Phase 1/2 trials for the treatment of solid tumors and lymphomas; Felzartamab, an antibody directed against CD38 for renal autoimmune diseases and relapsed/refractory multiple myeloma; Ianalumab, a candidate in Phase 3 clinical trials for Sjögren's disease, lupus nephritis, and other autoimmune diseases; Abelacimab that is in Phase 3 trials for venous thromboembolism prevention and cancer-associated thrombosis; Setrusumab, which is in Phase 2/3 trials for osteogenesis imperfecta; and Bimagrumab, a product candidate in Phase 2b trials for adult obesity. It also develops MOR210/TJ210/HIB210 that is in Phase 1 clinical trials for relapsed or refractory advanced solid tumors; and NOV-8, a candidate in Phase 2 trials for the treatment of pulmonary sarcoidosis and dermatitis. The company has collaboration and licensing agreements with I-Mab Biopharma, Novartis, Anthos Therapeutics, Ultragenyx, Mereo BioPharma, Lilly, Human Immunology Biosciences, Inc. Incyte Corporation, and Xencor, Inc. MorphoSys AG was founded in 1992 and is headquartered in Planegg, Germany.
POINT Biopharma Global
NASDAQ:PNTPOINT Biopharma Global Inc., a radiopharmaceutical company, develops and commercializes radioligands that fight cancer. Its lead product candidates include PNT2002, a prostate-specific membrane antigen (PSMA) targeted radioligand that is in Phase III trial for the treatment of metastatic castration-resistant prostate cancer; and PNT2003, a somatostatin-targeted radioligand, which is in Phase III trial for the treatment of neuroendocrine tumors. The company is also developing PNT2001, a next-generation PSMA-targeting product candidate for the treatment of prostate cancer, which is under preclinical studies; and PNT-2004, a fibroblast activation protein-alpha inhibitor targeting program being developed for use in various tumor types that is under preclinical studies. In addition, it has product candidates being developed on CanSEEK technology sub-licensed from both Bach Biosciences LLC and Avacta Life Sciences Limited. POINT Biopharma Global Inc. was founded in 2019 and is headquartered in Indianapolis, Indiana.
Avidity Biosciences
NASDAQ:RNAAvidity Biosciences, Inc., a biopharmaceutical company, engages in the delivery of RNA therapeutics. It develops antibody oligonucleotide conjugates (AOC) that are designed to treat diseases previously untreatable with RNA therapeutics. The company's lead product candidate AOC 1001 for the treatment of myotonic dystrophy type 1, a rare monogenic muscle disease that is in phase 1/2 clinical trial. Its other products in pipeline include AOC 1044 for the treatment of duchenne muscular dystrophy, which is under phase 1/2 clinical development trial; and AOC 1020 to treat facioscapulohumeral muscular dystrophy that is in phase 1/2 clinical trial. The company is developing products for rare skeletal muscle and rare cardiac diseases. Avidity Biosciences, Inc. was incorporated in 2012 and is headquartered in San Diego, California.
TG Therapeutics
NASDAQ:TGTXTG Therapeutics, Inc., a commercial stage biopharmaceutical company, focuses on the acquisition, development, and commercialization of novel treatments for B-cell mediated diseases in the United States and internationally. It provides BRIUMVI, an anti-CD20 monoclonal antibody for the treatment of adult patients with relapsing forms of multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease in adults. The company's development pipeline comprises Umbralisib, an oral inhibitor of PI3K-delta inhibitor for the treatment of adult patients with relapsed or refractory marginal zone lymphoma and follicular lymphoma; TG-1701 is an orally available and covalently bound Bruton's tyrosine kinase (BTK) inhibitor that exhibits selectivity to BTK in vitro kinase screening; and TG-1801, a bispecific CD47 and CD19 antibody. Its research pipeline includes various investigational medicines. The company has license agreements with LFB Biotechnologies S.A.S; GTC Biotherapeutics; LFB/GTC LLC; Ildong Pharmaceutical Co. Ltd.; Rhizen Pharmaceuticals, S A. for the development and commercialization of umbralisib; Jiangsu Hengrui Medicine Co.; and Novimmune SA, as well as collaboration agreement with Checkpoint Therapeutics, Inc. for the development and commercialization of anti-PD-L1 and anti-GITR antibody research programs in the field of hematological malignancies. TG Therapeutics, Inc. was incorporated in 1993 and is based in Morrisville, North Carolina.