4D Molecular Therapeutics, Inc. Common Stock
4D Molecular Therapeutics, Inc., a late-stage biotechnology company, engages in the development of adeno-associated virus vectors from its proprietary synthetic vector discovery platform, Therapeutic Vector Evolution in the Netherlands and the United States. Its lead product candidate is 4D-150 for the treatment of retinal vascular diseases by providing multi-year sustained production of anti-VEGF from the retina with intravitreal injection, as well as for treating wet age-related macular degeneration and diabetic macular edema. The company is also developing 4D-710, which is in early-stage study for the treatment of cystic fibrosis; 4D-175 that is in in preclinical stage for treating geographic atrophy; 4D-725, which is in preclinical stage for the treatment of alpha-1 anti-trypsin deficiency lung disease; and 4D-310 that is in Phase 1 clinical trial for treating Fabry disease. 4D Molecular Therapeutics, Inc. has collaboration and licensing agreements with Astellas Gene Therapies, Inc.; Arbor Biotechnologies, Inc.; and uniQure biopharma B.V. The company was founded in 2013 and is headquartered in Emeryville, California.
Open FDMT 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 ↗