Alnylam Pharmaceuticals, Inc. (ALNY)

業務介紹

Alnylam Pharmaceuticals, Inc.（艾尼藍制藥有限公司）是一家生物製藥公司，專注於基於核糖核酸干擾的新型治療藥物的發現、開發和商業化。該公司的RNAi治療藥物管道主要集中在遺傳性疾病、心血管代謝疾病、肝臟感染性疾病和中樞神經系統/眼科疾病方面。其市場產品包括ONPATTRO（帕替西蘭），一種用於治療成年人遺傳性甲狀腺激素介導的淀粉樣蛋白沉積症的多發性神經病變的脂質複合物注射劑；GIVLAARI用於治療成年人急性肝性卟啉症（AHP）；以及OXLUMO（盧馬西蘭）用於治療一型原發性高草酸尿症（PH1）。此外，該公司還正在開發GIVOSIRAN用於治療青少年患者的AHP；帕替西蘭用於治療甲狀腺激素介導的淀粉樣蛋白沉積症，或稱ATTR淀粉樣蛋白沉積症，伴有心肌病；CEMDISIRAN用於治療補體介導的疾病；ALN-AAT02用於治療與AAT缺乏相關的肝臟疾病；ALN-HBV02用於治療慢性乙型肝炎感染；ZILEBESIRAN用於治療高血壓；以及ALN-HSD用於治療非酒精性脂肪性肝炎。此外，該公司還提供FITUSIRAN用於治療血友病和罕見出血性疾病，INCLISIRAN用於治療高膽固醇血症，LUMASIRAN用於治療進展期PH1和反復腎結石，以及VUTRISIRAN用於治療ATTR淀粉樣蛋白沉積症，目前正在進行3期臨床試驗。Alnylam Pharmaceuticals, Inc.與Regeneron Pharmaceuticals, Inc.建立了戰略合作關係，以發現、開發和商業化RNAi治療藥物，針對眼睛和中樞神經系統中的治療靶點；並與Sanofi Genzyme建立了合作關係，以發現、開發和商業化RNAi治療藥物。該公司還與Novartis AG、Vir Biotechnology, Inc.、Dicerna Pharmaceuticals, Inc.、Ionis Pharmaceuticals, Inc.和PeptiDream, Inc.簽訂了許可和合作協議。該公司成立於2002年，總部位於麻薩諸塞州劍橋市。

Alnylam Pharmaceuticals, Inc., a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company's pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS)/ocular diseases. Its marketed products include ONPATTRO (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria (AHP); and OXLUMO (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1). In addition, the company is developing givosiran for the treatment of adolescent patients with AHP; patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; ALN-AAT02 for the treatment of AAT deficiency-associated liver disease; ALN-HBV02 to treat chronic HBV infection; Zilebesiran to treat hypertension; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia and rare bleeding disorders, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1 and recurrent renal stones, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals, Inc. has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Sanofi Genzyme to discover, develop, and commercialize RNAi therapeutics. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

公司網址

https://www.alnylam.com