Rapport Therapeutics, Inc. Common Stock
Rapport Therapeutics, Inc., operates as a clinical-stage biopharmaceutical company that focuses on the discovery and development of transformational small molecule medicines for patients suffering from central nervous system (CNS) disorders. Its lead product candidate is receptor associated protein (RAP)-219, an investigational small molecule that is designed to inhibit TARPy8-containing AMPARs with picomolar affinity for the treatment of focal epilepsy and other CNS disorders, including peripheral neuropathic pain and bipolar disorder. The company also develops RAP-199, a TARPy8 targeted molecule with differentiated chemical and pharmacokinetic properties; and nicotinic acetylcholine receptor (nAChR) programs, such as a6 nAChR to treat chronic pain and a9a10 nAChR for the treatment of hearing disorders. The company was formerly known as Precision Neuroscience NewCo, Inc. and changed its name to Rapport Therapeutics, Inc. in October 2022. Rapport Therapeutics, Inc. was incorporated in 2022 and is based in Boston, Massachusetts.
Open RAPP 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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