울트라제닉스 파마슈티컬 (RARE) 주가 전망: 목표주가, 배당, 최신 뉴스

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.

Ultragenyx Pharmaceutical Inc.는 희귀·초희귀 유전질환 치료를 위한 신약의 발굴·인수·개발·상용화에 주력하는 바이오제약 회사로, 북미·라틴아메리카·유럽·중동·아프리카·아시아·태평양 지역에서 활동합니다.

주요 바이오 제품으로는 X-연관 저인산혈증(X-linked hypophosphatemia) 및 종양유발 골연화증 치료를 위한 섬유아세포성장인자23(FGF23)을 표적하는 항체 Crysvita (burosumab), MPS VII(뮤코다당증 VII) 아동 및 성인 치료를 위한 효소대체요법 Mepsevii, 장쇄 지방산 산화장애 치료제 Dojolvi, 그리고 동형접합 가족성 고콜레스테롤혈증 치료제 Evkeeza (evinacumab)가 있습니다.

임상 파이프라인으로는 임상 3상에 있는 골형성부전 치료용 휴먼 단일클론 항체 UX143, AAV9 유전자치료 후보 UX111(샌필리포 증후군 A형/MPS IIIA), AAV8 유전자치료 후보 DTX401(글리코겐 저장병 Ia형), AAV8 유전자치료 DTX301(오르니틴 트랜스카바밀레이스 결핍증), 안티센스 올리고뉴클레오타이드 GTX-102(앵글맨 증후군) 등이 있으며, 임상 2상에서는 윌슨병 치료제 UX701와 글리코겐 저장병 III형 치료제 UX053을 개발 중입니다. 회사는 Kyowa Kirin, Saint Louis University, Baylor Research Institute, REGENXBIO, GeneTx, Mereo, University of Pennsylvania, Solid Biosciences, Regeneron, Abeona, Arcturus Therapeutics 등과 협력·라이선스 계약을 맺고 있습니다.

회사는 2010년에 설립되었으며 본사는 캘리포니아주 노바토에 있습니다.

• 주소: 60 Leveroni Court, Novato, CA 94949, United States

• 전화번호: 415 483 8800

• 웹사이트: https://www.ultragenyx.com

• 섹터: Healthcare

• 산업: Biotechnology

• 직원 수: 1,371