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由诺贝尔化学奖得主 Jennifer Doudna 共同创办的 Mammoth Biosciences（Mammoth）日前宣布募得 1.95 亿美元融资，成功晋升生技独角兽的行列。
1.95 亿美元分别来自两次融资轮
独角兽是业界指称创立不到 10 年，但估值突破 10 亿美元的未上市公司。Mammoth 自 2017 年创立后，透过多项 CRISPR 产品吸引投资人目光，而这 1.95 亿美元是由 D 轮融资的 1.5 亿美元与 C 轮融资的 4,500 万美元组成。2 场融资轮次皆由美国 Redmile Group 领投，投资者也包含 Foresite Capital、Amazon、Sixth Street 与 Senator Investment Group 等。
Mammoth 基因编辑军火库：2 款超小型 Cas 编辑蛋白
CRISPR 基因编辑技术是靠 Cas9 蛋白进行 DNA 编辑，去除错误基因，从根本治疗病症，除了 Cas9 之外，也有其他可编辑 DNA 的工具。在这数年内，Mammoth 开发了不少编辑工具，目前有 Cas12、Cas13、Cas14、Casɸ 四款，其中 Cas14 与 Casɸ 是目前最小的基因编辑蛋白之一，因为可轻松置入腺病毒载体中传递，所以使体内编辑与治疗更容易。另外，Mammoth 其他的编辑工具也具备特定优势，譬如标靶范围广，或在高温下稳定性高等。
除了治疗面向，Mammoth 也将 CRISPR 技术投入检测技术中。该公司在 COVID-19 疫情下第 1 个推出 CRISPR 新冠病毒检测技术，随后也与葛兰素史克（GSK）、美国国卫院（NIH）与美国国防高等研究计划署（DARPA）进行合作。
Jennifer Doudna 其他新创：Intellia 完成体内 CRISPR 编辑临床试验
Jennifer Doudna 不只创立 Mammoth 一家新创公司，CRISPR Therapeutics 与 Intellia Therapeutics（Intellia）也是她以 CRISPR 为出发点所创立的公司。
近期 Intellia 在今年 6 月底出现突破性进展。该公司与再生元制药（Regeneron Pharmaceuticals）完成全球第 1 个 CRISPR 体内编辑的第 1 期临床试验。
该试验标靶转甲状腺蛋白类淀粉沉积症（ATTR），一共招募 6 位受试者，标靶他们的肝细胞 TTR 基因。结果指出，该疗法大幅降低患者体内异常沈淀的 TTR 蛋白浓度，0.3 mg/kg 组在用药 28 天后，TTR 蛋白浓度平均降低 87%。
延伸阅读：两项专利助攻！ Mammoth Biosciences开发第一个CRISPR疾病检测平台©www.geneonline.news. All rights reserved. 基因线上版权所有 未经授权不得转载。合作请联系：service@geneonlineasia.com
