Monte Rosa Therapeutics, Inc. Common Stock
Monte Rosa Therapeutics, Inc., a clinical-stage biotechnology company, engages in the development of novel small molecule precision medicines that employ the body's natural mechanisms to selectively degrade therapeutically relevant proteins. The company develops orally bioavailable molecular glue degraders (MGDs), such as MRT-2359 for the treatment of MYC-driven tumors; MRT-6160 for the treatment of neurologic and systemic autoimmune and inflammatory diseases; and MRT-8102 for the treatment of inflammatory diseases driven by IL-1b and the NLRP3 inflammasome. In addition, it develops CDK2 to treat multiple cancers, including ovarian, endometrial, gastric and breast. Further, its pipeline includes programs in immunology and inflammation indications, oncology, cardiovascular, metabolic, and genetic diseases. The company also develops QuEEN, a discovery engine, to enable its target-centric MGD discovery and development approach and its rational design of MGD product candidates. It has a strategic collaboration and licensing agreement with F. Hoffmann-La Roche Ltd. to discover and develop MGDs against targets in cancer and neurological diseases. The company was incorporated in 2019 and is headquartered in Boston, Massachusetts.
Open GLUE 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.
Open RARE dashboard ↗