Lexeo Therapeutics, Inc. Common Stock
Lexeo Therapeutics, Inc., a clinical stage genetic medicine company, focuses on hereditary and acquired diseases with high unmet need in the United States. Its product pipeline comprises LX2006, an AAVrh10-based gene therapy candidate, which is in phase 1/2 clinical trial to treat friedreich ataxia cardiomyopathy; LX2020, an AAVrh10-based gene therapy candidate, which is in phase 1/2 to deliver a functional plakophilin-2 (PKP2) gene to cardiac muscle for the treatment of PKP2-ACM; and LX2021, a gene therapy candidate, which is in preclinical trial to deliver the coding sequence for the functional connexin 43, or Cx43, protein for a group of inherited cardiac muscle disorders associated with a high risk of sudden death, including arrhythmogenic cardiomyopathy (ACM) and certain forms of dilated cardiomyopathy. The company also develops LX2022, a gene therapy candidate, which is in preclinical trial to deliver a functional TNNI3 gene to myocardial cells to treat a distinct form of hypertrophic cardiomyopathy. In addition, it is developing LX1001, an AAVrh10-based gene therapy candidate, which has completed phase 1/2 clinical trial for the treatment of APOE4-associated Alzheimer's disease. The company has license agreement with Cornell University to conduct of the Phase 1/2 clinical trial of LX1001, as well as support the development of LX1004 program; research collaboration agreement with Cornell University to conduct preclinical research to develop the licensed technology; and third license agreement which obtained certain rights for FA cardiomyopathy, including rights to current and future clinical data from an ongoing Cornell University investigator-initiated Phase 1A trial of a gene therapy candidate. The company was founded in 2017 and is headquartered in New York, New York.
Open LXEO 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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