Krystal Biotech, Inc. Common Stock
Krystal Biotech, Inc., a commercial-stage biotechnology company, discovers, develops, manufactures, and commercializes genetic medicines to treat diseases with high unmet medical needs in the United States. The company commercializes VYJUVEK (beremagene geperpavec-svdt, or B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB). It also develops KB105, which is in Phase 1/2 clinical trials for treating patients with deficient autosomal recessive congenital ichthyosis; KB104 for treating netherton syndrome; KB407 that is in Phase 1 clinical trials for treating cystic fibrosis; KB707 that is in Phase 1/2 clinical trials for the treatment of anti-PD-1 relapsed/refractory; KB408, which is in Phase 1 clinical trials for treating Alpha-1 antitrypsin deficiency; and KB301 that is in Phase 2 clinical trials for treating aesthetic skin conditions, as well as in open label study with ophthalmic B-VEC for treating for ocular complications of deb. Krystal Biotech, Inc. was founded in 2016 and is headquartered in Pittsburgh, Pennsylvania.
Open KRYS 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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