Korro Bio, Inc. Common Stock
Korro Bio, Inc., a biopharmaceutical company, engages in the discovering, developing, and commercializing of genetic medicines based on editing RNA for the treatment of rare and highly prevalent diseases in the United States. Its lead product candidate is KRRO-110 which is in Phase 1/2 clinical trial for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). It uses its RNA editing platform, Oligonucleotide Promoted Editing of RNA to generate differentiated RNA editing product candidates. The company is also developing programs for Parkinson's disease, Amyotrophic lateral sclerosis, severe drug-associated anesthetics, and other pain indications. Korro Bio, Inc. has a collaboration agreement with Novo Nordisk A/S to treat cardiometabolic diseases. Korro Bio, Inc. was founded in 2018 and is based in Cambridge, Massachusetts.
Open KRRO dashboard ↗Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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