Precision BioSciences, Inc. Common Stock
Precision BioSciences, Inc., a clinical stage gene editing company, develops in vivo gene editing therapies for gene edits, including gene insertion, excision, and elimination in the United States. The company offers ARCUS, a genome editing platform for DNA genome insertion, removal, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits, as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; and PBGENE-3243 for the treatment of m.3243 associated mitochondrial myopathy. In addition, the company develops PBGENE-NVS (insertion) for sickle cell and beta thalassemia diseases; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LIVER (insertion), a liver directed target; PBGENE-CNS (excision), a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. It has collaboration agreements with Novartis Pharma AG to discover and develop in vivo gene editing products; TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; and iECURE, Inc. to develop ARCUS-based gene-insertion therapies. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.
Open DTIL 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.
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