4D Molecular Therapeutics, Inc. Common Stock
4D Molecular Therapeutics, Inc., a late-stage biotechnology company, engages in the development of adeno-associated virus vectors from its proprietary synthetic vector discovery platform, Therapeutic Vector Evolution in the Netherlands and the United States. Its lead product candidate is 4D-150 for the treatment of retinal vascular diseases by providing multi-year sustained production of anti-VEGF from the retina with intravitreal injection, as well as for treating wet age-related macular degeneration and diabetic macular edema. The company is also developing 4D-710, which is in early-stage study for the treatment of cystic fibrosis; 4D-175 that is in in preclinical stage for treating geographic atrophy; 4D-725, which is in preclinical stage for the treatment of alpha-1 anti-trypsin deficiency lung disease; and 4D-310 that is in Phase 1 clinical trial for treating Fabry disease. 4D Molecular Therapeutics, Inc. has collaboration and licensing agreements with Astellas Gene Therapies, Inc.; Arbor Biotechnologies, Inc.; and uniQure biopharma B.V. The company was founded in 2013 and is headquartered in Emeryville, California.
Open FDMT 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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