C4 Therapeutics, Inc.
C4 Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops novel therapeutic candidates to degrade disease-causing proteins. Its lead product candidate is Cemsidomide, an orally bioavailable MonoDAC degrader of protein that is in Phase 1/2 clinical trials targeting IKZF1 and IKZF3 for multiple myeloma and non-Hodgkin lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma. The company is also developing CFT1946, an orally bioavailable BiDAC degrader targeting BRAF V600 mutant that is in Phase 1/2 clinical trials to treat melanoma, colorectal cancer, and other solid malignancies; and CFT8919, an orally bioavailable, allosteric, and mutant-selective BiDAC degrader of epidermal growth factor receptor with an L858R mutation in non-small cell lung cancer. It has strategic collaborations with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; Biogen MA, Inc.; Betta Pharmaceuticals, Co., Ltd.; Merck KGaA; and Merck Sharp & Dohme, LLC, as well as Calico Life Sciences LLC. The company was incorporated in 2015 and is headquartered in Watertown, Massachusetts.
Open CCCC dashboard ↗Rocket Pharmaceuticals, Inc. Common Stock
Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). 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; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
Open RCKT dashboard ↗