Precision BioSciences, Inc. Common Stock
Precision BioSciences, Inc., a clinical stage gene editing company, develops in vivo gene editing therapies for gene edits, including gene insertion, excision, and elimination in the United States. The company offers ARCUS, a genome editing platform for DNA genome insertion, removal, and repair. It also provides PBGENE-HBV for the treatment of chronic hepatitis B virus (HBV) to eliminate covalently closed circular DNA with direct cuts and edits, as well as to inactivate integrated HBV DNA with the goal of long-lasting reductions in hepatitis B surface antigen; and PBGENE-3243 for the treatment of m.3243 associated mitochondrial myopathy. In addition, the company develops PBGENE-NVS (insertion) for sickle cell and beta thalassemia diseases; PBGENE-DMD (excision) for duchenne muscular dystrophy; PBGENE-LIVER (insertion), a liver directed target; PBGENE-CNS (excision), a central nervous system directed target; and iECURE-OTC (insertion) for ornithine transcarbamylase deficiency. It has collaboration agreements with Novartis Pharma AG to discover and develop in vivo gene editing products; TG Cell Therapy, Inc. to develop, manufacture, and commercialize azer-cel for autoimmune diseases and other indications outside of cancer; and iECURE, Inc. to develop ARCUS-based gene-insertion therapies. Precision BioSciences, Inc. was incorporated in 2006 and is headquartered in Durham, North Carolina.
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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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