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BHVN vs RARE

Biohaven Ltd. and Ultragenyx Pharmaceutical Inc.

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Biohaven Ltd.

Biohaven Ltd. discovers, develops, and commercializes therapies for immunology, neuroscience, and oncology worldwide. The company develops troriluzole, which is in Phase 3 trial for the treatment of neurological and neuropsychiatric illnesses; taldefgrobep alfa, which is in Phase 3 clinical trial for the treatment of spinal muscular atrophy and obesity; BHV-7000, a candidate in Phase 3 clinical trial for the treatment of focal and generalized epilepsy, and major depressive disorder; BHV-2100 that is in Phase 2 clinical trial for the treatment of migraines and pain disorders; BHV-1510, which is in Phase ½ trial that targets advanced or metastatic epithelial tumors; and BHV-8000, a product candidate that completed Phase 1 clinical trial for the treatment of Alzheimer's and Parkinson's disease, and multiple sclerosis, as well as prevention of amyloid-related imaging abnormalities. It is also developing BHV-1300, which in Phase 1 clinical trial to treat Graves' disease and rheumatoid arthritis; BHV-1400 that is in Phase 1 trial for IgA nephropathy; BHV-1100, which is in Phase 1 trial for multiple myeloma patients; BHV-1600, which is in Phase 1 trial for the treatment of peripartum cardiomyopathy; BHV-1310 that is in preclinical trial for the treatment of generalized myasthenia gravis and other acute exacerbations or flares; BHV-1530, which is in preclinical trial to treat urothelial cancer; BHV-1500 that is in preclinical trial for Hodgkin's lymphoma; and BHV-5500 that blocks glutamate signaling mediated by post-synaptic NMDA receptors. The company has strategic partnerships with Merus N.V., GeneQuantum Healthcare (Suzhou) Co. Ltd., Aimed Bio, Inc., Yale University, Katholieke Universiteit Leuven, Bristol Meyers Squibb, and Hangzhou Highlightll Pharmaceutical Co. Ltd. Biohaven Ltd. was formerly known as Biohaven Research Ltd. and changed its name to Biohaven Ltd. in September 2022. Biohaven Ltd. was incorporated in 2022 and is headquartered in New Haven, Connecticut.

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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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