Intellia Therapeutics
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About Intellia Therapeutics
Intellia Therapeutics, Inc. 是一家基因組編輯公司，專注於治療學的開發。該公司的體內項目包括 NTLA-2001，該藥物正處於治療轉甲狀腺素蛋白澱粉樣變性的 1 期臨床試驗階段；和 NTLA-2002 用於治療遺傳性血管性水腫，以及其他以肝臟為中心的項目，包括血友病 A 和血友病 B、1 型高草酸尿症和 alpha-1 抗胰蛋白酶缺乏症。其體外管線包括用於治療急性髓性白血病的NTLA-5001；和專有項目專注於開發工程細胞療法來治療各種腫瘤和自身免疫性疾病。此外，它還提供由成簇、規律間隔的短回文重複序列/CRISPR 相關 9 (CRISPR/Cas9) 系統組成的工具。 Intellia Therapeutics, Inc. 與 Novartis Institutes for BioMedical Research, Inc. 簽訂了許可和合作協議，以設計造血幹細胞來治療鐮狀細胞病； Regeneron Pharmaceuticals, Inc. 共同開發治療血友病 A 和血友病 B 的潛在產品； Ospedale 聖拉斐爾；與 SparingVision SAS 的戰略合作，利用 CRISPR/Cas9 技術開發新型基因組藥物，用於治療眼部疾病。該公司前身為 AZRN , Inc . Intellia Therapeutics , Inc .成立於2014年，總部位於馬薩諸塞州劍橋市。
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