Disc Medicine, Inc. Common Stock
Disc Medicine, Inc., together with its subsidiaries, a clinical-stage biopharmaceutical company, engages in the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases in the United States. The company has assembled a portfolio of clinical and preclinical product candidates that aim to modify fundamental biological pathways associated with the formation and function of red blood cells, primarily heme biosynthesis and iron homeostasis. Its pipeline includes bitopertin for the treatment of erythropoietic porphyrias, including erythropoietic protoporphyria, X-linked protoporphyria, and diamond-blackfan anemia; DISC-0974 for the treatment of anemia of myelofibrosis, and anemia of chronic kidney disease; and DISC-3405 for the treatment of polycythemia vera, and other hematologic disorders. It also develops preclinical programs include DISC-0998, for the treatment of anemia associated with inflammatory diseases. Disc Medicine, Inc. was founded in 2017 and is headquartered in Watertown, Massachusetts.
Open IRON 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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