Intellia Therapeutics, Inc
Intellia Therapeutics, Inc., a clinical-stage gene editing company, focuses on the development of curative genome editing treatments. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 that is in Phase 1/2 study for the treatment of hereditary angioedema. It also focusses on the research of proprietary programs focused on developing engineered cell therapies to treat various cancers and autoimmune diseases. The company has license and collaboration agreement with AvenCell Therapeutics, Inc. to develop allogeneic universal CAR-T cell therapies; Kyverna Therapeutics, Inc. for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of various of B cell-mediated autoimmune diseases; ONK Therapeutics, Ltd. for the development of engineered NK cell therapies to cure patients with cancer; and ReCode Therapeutics, Inc. to develop novel genomic medicines for the treatment of cystic fibrosis. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.
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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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