Sarepta Therapeutics, Inc. (SRPT)

業務介紹

Sarepta Therapeutics, Inc.是一家商業化階段的生物製藥公司，專注於發現和開發針對RNA的治療方法、基因療法和其他基因治療模式，用於治療罕見疾病。該公司提供EXONDYS 51注射劑，用於治療杜氏肌肉萎縮症（杜氏症），適用於已確認患有可進行exon 51跳過的dystrophin基因突變的患者；以及VYONDYS 53，用於治療杜氏症，適用於已確認患有可進行exon 53跳過的dystrophin基因突變的患者。該公司還正在開發AMONDYS 45，一種使用磷酸二胺基甲酸酯基態和exon跳過技術來跳過dystrophin基因exon 45的產品候選藥物；SRP-5051，一種與dystrophin前mRNA的exon 51結合的肽共軛PMO；SRP-9001，一個DMD微小肌肉萎縮基因治療計劃；以及SRP-9003，一個四肢帶狀肌肉萎縮基因治療計劃。該公司與F. Hoffman-La Roche Ltd、Nationwide Children's Hospital、Lysogene、Duke University、Genethon和StrideBio簽訂了合作協議。該公司成立於1980年，總部位於麻薩諸塞州劍橋市。

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; and VYONDYS 53 for the treatment of duchenne in patients with confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. The company is also developing AMONDYS 45, a product candidate that uses phosphorodiamidate morpholino oligomer chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene; SRP-5051, a peptide conjugated PMO that binds exon 51 of dystrophin pre-mRNA; SRP-9001, a DMD micro-dystrophin gene therapy program; and SRP-9003, a limb-girdle muscular dystrophies gene therapy program. It has collaboration agreements with F. Hoffman-La Roche Ltd; Nationwide Children's Hospital; Lysogene; Duke University; Genethon; and StrideBio. The company was incorporated in 1980 and is headquartered in Cambridge, Massachusetts.

公司網址

https://www.sarepta.com