Passage Bio, Inc. Common Stock
Passage Bio, Inc., a genetic medicines company, develops gene therapies for central nervous system diseases. It develops PBGM01, a functional GLB1 gene encoding ß-galactosidase for infantile GM1; PBFT02, a functional granulin (GRN) and gene encoding progranulin (PGRN) for the treatment of FTD caused by progranulin deficiency; and PBKR03, a functional GALC gene encoding the hydrolytic enzyme galactosylceramidase for infantile Krabbe disease. The company develops PBML04 for the treatment of metachromatic leukodystrophy; PBAL05 for the treatment of amyotrophic lateral sclerosis; and other program for huntington's disease. It has a strategic research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program; and collaboration agreement, and a development services and clinical supply agreement with Catalent Maryland, Inc. The company was incorporated in 2017 and is based in Philadelphia, Pennsylvania.
Open PASG 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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