treatments capable of precisely and durably engineering the hematopoietic system, today announced the U.S. Food and Drug Administration (FDA) has granted both rare pediatric disease and orphan drug designations to the company’s lead program, EN-374, for the treatment of X-linked chronic granulomatous disease (X-CGD).

Ensoma 是一家基因组药物公司，致力于通过一次性体内治疗来精确持久地改造造血系统，推动医学的未来发展。该公司今天宣布，美国食品药品管理局 (FDA) 已授予该公司的领先项目 EN-374 罕见儿科疾病和孤儿药资格，用于治疗 X 连锁慢性肉芽肿病 (X-CGD)。Ensoma 预计将在 2025 年上半年提交 EN-374 的新药临床试验申请 (IND)。

Chronic granulomatous disease (CGD) is a rare and severe genetic disorder, which significantly impairs the immune system. It leaves patients vulnerable to recurrent and severe bacterial and fungal infections, often leading to chronic and life-threatening dysregulated inflammation and serious complications.

慢性肉芽肿性疾病（CGD）是一种罕见且严重的遗传疾病，会严重损害免疫系统。它使患者容易反复出现严重的细菌和真菌感染，通常会导致慢性和危及生命的炎症失调和严重并发症。

CGD affects approximately 1 in 100,000-200,000 live births, and the median life expectancy for individuals with the condition is around 45 years. X-CGD is the most common form of the condition, affecting 60-70% of CGD patients, and is caused by mutations in the .

CGD影响大约100000-200000例活产中的1例，患有这种疾病的个体的平均预期寿命约为45岁。。

gene that prevent white blood cells called neutrophils from fighting infection. EN-374 is designed to treat X-CGD through the delivery of a functional

阻止称为中性粒细胞的白细胞抵抗感染的基因。EN-374旨在通过传递功能来治疗X-CGD

gene directly to hematopoietic stem cell (HSCs)

基因直接进入造血干细胞（HSC）

. It uses a promoter for selective expression in neutrophils, the primary cell type affected by X-CGD. Successful expression will restore patients’ ability to fight infection.

它使用启动子在中性粒细胞中选择性表达，中性粒细胞是受X-CGD影响的原代细胞类型。成功表达将恢复患者抵抗感染的能力。

“These designations from the FDA highlight the significant medical need in chronic granulomatous disease,” said Drew Dietz, M.D., MSCR, vice president of clinical development at Ensoma. “Current treatments, including antibiotics, antifungals, interferon gamma and allogeneic stem cell transplantation, offer limited benefit and/or come with significant burdens.

Ensoma临床开发副总裁DrewDietz医学博士说：“FDA的这些名称突显了慢性肉芽肿疾病的重大医疗需求。”。“目前的治疗方法，包括抗生素、抗真菌药、干扰素-γ和异基因干细胞移植，效益有限和/或负担沉重。

EN-374 represents the first .

EN-374代表第一个。

HSC-directed therapy for X-CGD, building on a mechanism that has been validated

HSC指导的X-CGD治疗，建立在已验证的机制之上

. Designed to function for any CYBB mutation, this approach offers the potential to improve upon transformative benefits of

。该方法旨在针对任何CYBB突变发挥作用，具有改善

gene therapies in a way that’s simpler, potentially safer and more accessible for patients. Our team is incredibly proud to be at the forefront of advancing this groundbreaking therapy. We remain on track to submit an IND in the first half of this year.”

基因治疗的方式更简单，可能更安全，患者更容易获得。我们的团队非常自豪能够走在这一开创性疗法的前沿。

Rare pediatric disease designation is granted by the FDA to incentivize development of treatments for serious or life-threatening rare diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the U.S. If a Biologics License Application for EN-374 is approved in the U.S., Ensoma may be eligible to receive a Priority Review Voucher from the FDA..

美国食品和药物管理局（FDA）授予罕见儿科疾病名称，以鼓励开发严重或危及生命的罕见疾病的治疗方法，这些疾病主要影响18岁或以下的儿童，影响美国不到20万人。如果EN-374的生物制剂许可证申请在美国获得批准，Ensoma可能有资格获得FDA的优先审查凭证。。

Orphan drug designation is granted by the FDA to products that prevent, diagnose or treat a rare disease or condition affecting fewer than 200,000 people in the U.S. The designation affords Ensoma the potential for certain benefits, including up to seven years market exclusivity after approval, tax credits for qualified clinical trials and exemption from certain FDA fees..

美国食品和药物管理局（FDA）授予孤儿药名称，用于预防、诊断或治疗影响美国不到20万人的罕见疾病或病症。该名称赋予恩索玛某些益处的潜力，包括批准后长达七年的市场排他性，合格临床试验的税收抵免以及免除某些FDA费用。。

About Ensoma

关于Ensoma

Ensoma is developing curative medicines through precision

恩索玛正在通过精准开发治疗药物

cellular engineering. Our platform combines class-leading proprietary base editing and high-efficiency gene integration systems with high-capacity virus-like particles (VLPs) to provide one-time and durable genetic medicines in an outpatient procedure. We are focused on

细胞工程。我们的平台将一流的专有基础编辑和高效基因整合系统与高容量病毒样颗粒（VLP）相结合，在门诊手术中提供一次性耐用的基因药物。我们专注于

engineering of hematopoietic stem cells (HSCs) to address genetic diseases, immune disorders and cancer. Ensoma is supported by top-tier investors and a passionate team committed to a bold, global vision for genomic medicines. Ensoma is based in Boston. For more information, visit

造血干细胞（HSC）工程，以解决遗传疾病，免疫疾病和癌症。Ensoma得到了顶级投资者和一支充满激情的团队的支持，他们致力于为基因组药物提供大胆的全球愿景。Ensoma总部位于波士顿。