Findings demonstrate ability of PM359 to correct disease-causing mutation in CGD patient blood stem cells, leading to restored immune function in vivo with no off-target edits detected IND for PM359 recently cleared by U.S. FDA; data support advancement into Phase 1/2 clinical trial CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc.

研究结果表明，PM359能够纠正CGD患者血液干细胞中的致病突变，从而恢复体内免疫功能，而美国FDA最近批准的PM359的IND没有检测到脱靶编辑；数据支持进入1/2期临床试验，剑桥，马萨诸塞州，2024年5月8日（环球通讯社）——Prime Medicine，Inc。

(Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of its ex vivo Prime Editing program, PM359, to correct a common disease-causing mutation of chronic granulomatous disease (CGD).

（纳斯达克：PRME），一家致力于提供一类新的分化的一次性治愈性基因疗法的生物技术公司，今天报道了新的临床前数据，证明了其离体初级编辑程序PM359纠正慢性肉芽肿性疾病（CGD）常见致病突变的能力。

The data will be presented today at an oral presentation during the American Society of Cell & Gene Therapy 27th Annual Meeting in Baltimore. Prime Medicine recently announced that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for PM359 for the treatment of CGD.

这些数据将于今天在巴尔的摩举行的美国细胞与基因治疗学会第27届年会上发表。Prime Medicine最近宣布，美国食品和药物管理局（FDA）已批准其用于治疗CGD的PM359研究性新药（IND）申请。

“These data are incredibly exciting—showing for the first time that Prime Editing can not only correct the disease-causing mutation of CGD in human blood stem cells, but that those cells can produce neutrophils with restored immune function and healthy activity following engraftment in rodents with no off-target edits observed,” said Jennifer Gori, Ph.D., Vice President, Head of Hematology and Immunology at Prime Medicine.

Prime Medicine副总裁兼血液学和免疫学负责人Jennifer Gori博士说：“这些数据令人难以置信地激动，首次表明Prime编辑不仅可以纠正人类造血干细胞中引起CGD突变的疾病，而且这些细胞可以在啮齿动物植入后产生免疫功能恢复和健康活动的中性粒细胞，没有观察到脱靶编辑。”。

“Further, we were able to demonstrate clinical-scale production of Prime Edited blood stem cells, supporting our planned advancement into the clinic with PM359.” CGD is a rare inherited disease that leads to recurrent, debilitating and often life-threatening infections. CGD is caused by mutations in any one of the subunits comprising the NADPH oxidase complex, an enzym.

“此外，我们能够证明初级编辑的血液干细胞的临床规模生产，支持我们计划用PM359进入临床。”CGD是一种罕见的遗传性疾病，会导致反复发作，使人衰弱，甚至危及生命的感染。CGD是由包含NADPH氧化酶复合物（一种酶）的任何一个亚基的突变引起的。