aTyr Pharma, Inc. Common Stock
aTyr Pharma, Inc., a clinical stage biotechnology company, engages in the discovery and development of product candidates that translate tRNA synthetase biology into new therapies for fibrosis and inflammation in the United States. Its lead therapeutic candidate is efzofitimod, a selective modulator of NRP2 that is in Phase 3 clinical trial for the treatment of pulmonary sarcoidosis; and in Phase 1b/2a clinical trial to treat other interstitial lung diseases (ILDs), such as chronic hypersensitivity pneumonitis and connective tissue disease related ILD. The company is developing ATYR0101, a fusion protein derived from a domain of aspartyl-tRNA synthetase, which is in preclinical development for the treatment fibrosis; and ATYR0750, a domain of alanyl-tRNA synthetase for the treatment of liver disorders. It has collaboration and license agreement with Kyorin Pharmaceutical Co., Ltd. for the development and commercialization of efzofitimod for ILDs in Japan. aTyr Pharma, Inc. was incorporated in 2005 and is headquartered in San Diego, California.
Open ATYR 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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