Biotech company Prime Medicine received a regulator’s green light to start testing its next-generation gene editing candidate in humans. On 29 April, the US Food and Drug Administration approved the biotech’s Investigational New Drug application, which gives Prime the go-ahead to test its first product candidate, PM359, in a phase 1/2 clinical trial in pediatric and adult patients with chronic granulomatosis disease (CGD).

CGD is a rare immunodeficiency caused by mutations in genes that affect phagocyte function by compromising nicotinamide adenine dinucleotide phosphate (NADPH) production and the cells’ ability to produce pathogen-killing reactive oxygen species. This renders patients susceptible to severe life-threatening bacterial and fungal infections. Hematopoietic stem cell transplants are the only definitive cure for CGD.

生物技术公司 Prime Medicine 获得监管机构批准，开始在人体中测试其下一代基因编辑候选药物。 4 月 29 日，美国食品和药物管理局批准了该生物技术公司的研究性新药申请，这使得 Prime 可以在患有慢性肉芽肿病的儿童和成人患者中进行 1/2 期临床试验，测试其第一个候选产品 PM359疾病（CGD）。

CGD 是一种罕见的免疫缺陷病，由基因突变引起，这些突变通过损害烟酰胺腺嘌呤二核苷酸磷酸 (NADPH) 的产生以及细胞产生杀死病原体的活性氧的能力来影响吞噬细胞功能。这使得患者容易受到严重危及生命的细菌和真菌感染。造血干细胞移植是治疗 CGD 的唯一确定方法。