Ionis Pharmaceuticals, Inc. (IONS)

業務介紹

艾尼斯製藥公司(Ionis Pharmaceuticals, Inc.)在美國發現和開發針對RNA的治療藥物。該公司提供以下產品:

1. SPINRAZA,用於治療小兒和成人脊髓性肌肉萎縮症(SMA)。
2. TEGSEDI,一種用於治療遺傳性轉甲狀腺素介導性淀粉樣變性多發性神經病變的注射劑。
3. WAYLIVRA,用於治療家族性乳糜微粒血症綜合症和家族性部分脂肪營養不良。

該公司還開發了多種適應症的藥物,正處於III期臨床試驗階段,包括:

1. Eplontersen,每月皮下自我注射一次,用於治療所有類型的轉甲狀腺素淀粉樣變性。
2. Olezarsen,用於嚴重高三酸甘油脂血症(SHTG)患者。
3. Donidalorsen,用於治療遺傳性血管性水腫患者。
4. ION363,用於治療肌萎縮性側索硬化症患者。
5. Pelacarsen,用於有既往心血管疾病且脂蛋白(a)升高的患者。
6. Tofersen,用於抑制超氧化物歧化酶1的產生。

此外,該公司還開發了用於代謝性疾病、感染性疾病、腎臟疾病、眼科疾病和癌症的藥物。該公司與Biogen Inc.有戰略合作關係,並與AstraZeneca、拜耳、葛蘭素史克、諾華、羅氏、強生生物技術和Flamingo Therapeutics, Inc.有合作和許可協議。艾尼斯製藥公司成立於1989年,總部位於加州卡爾斯巴德。



Ionis Pharmaceuticals, Inc. discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy. It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of TTR amyloidosis; Olezarsen for patients with severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Tofersen to inhibit the production of superoxide dismutase 1. In addition, the company develops medicines for metabolic diseases, infectious diseases, renal diseases, ophthalmic diseases, and cancer. It has a strategic collaboration with Biogen Inc.; and collaboration and license agreement with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.

公司網址

https://www.ionispharma.com