/PRNewswire/ -- SineuGene Therapeutics, a clinical-stage biotechnology company focused on gene therapies for neurological diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its gene therapy, SNUG01, for the treatment of amyotrophic lateral sclerosis (ALS)..

/PRNewswire/ -- 专注于神经系统疾病基因疗法的临床阶段生物技术公司SineuGene Therapeutics宣布，美国食品和药物管理局（FDA）已授予其基因疗法SNUG01用于治疗肌萎缩侧索硬化症（ALS）的孤儿药资格（ODD）。

SNUG01 is a first-in-class gene therapy targeting TRIM72 (Tripartite Motif Containing 72), designed to provide broad neuroprotection in ALS, a rare, fatal neurodegenerative disorder affecting approximately 30,000 individuals in

SNUG01 是一种针对 TRIM72（包含三部分的基序 72）的首创基因疗法，旨在为 ALS（肌萎缩侧索硬化症）提供广泛的神经保护，这是一种罕见的、致命的神经退行性疾病，大约影响 30,000 人。

FDA Orphan Drug Designation status is granted for treatments of rare diseases affecting fewer than 200,000 people in the U.S. This designation provides SineuGene with a range of development and commercial incentives, including tax credits for qualified clinical trials, exemption from BLA user fees, and eligibility for seven years of marketing exclusivity ('orphan exclusivity') upon approval..

FDA孤儿药资格状态授予用于治疗在美国影响少于20万人的罕见疾病的疗法。该资格为SineuGene提供了一系列开发和商业激励措施，包括针对合格临床试验的税收抵免、免除BLA用户费用，以及在获批后享有七年的市场独占权（“孤儿独占权”）。

The designation follows the FDA's clearance of the Investigational New Drug (IND) application for SNUG01 in

该指定是在FDA批准SNUG01的研究性新药（IND）申请之后进行的。

March 2025

2025年3月

. SineuGene plans to initiate a Phase I/IIa international, multicenter trial to evaluate the therapy's safety, tolerability, and preliminary efficacy in adults with ALS. The study will enroll participants at clinical sites across both countries, including Massachusetts General Hospital in the U.S., which will join other sites in .

SineuGene 计划启动一项国际多中心 I/IIa 期试验，以评估该疗法在 ALS 成人患者中的安全性、耐受性和初步疗效。该研究将在两国的临床试验点招募参与者，其中包括美国麻萨诸塞州综合医院，该医院将与其他试验点一起加入研究。

About SNUG01

关于SNUG01

SNUG01, developed by SineuGene via its proprietary AAV technology platform, is a first-in-class gene therapy product using TRIM72 as the gene of interest (GOI). The TRIM72 was identified as a potential therapeutic gene based on foundational research from Dr.

SNUG01是由SineuGene通过其专有的AAV技术平台开发的首例基因治疗产品，使用TRIM72作为目标基因（GOI）。TRIM72基于Dr.的基础研究被确定为潜在的治疗基因。

Yichang Jia's

宜昌佳的

lab at Tsinghua University. SNUG01 utilizes a rAAV9 vector to deliver the human TRIM72 gene to neurons via intrathecal administration. Preclinical studies have shown that TRIM72 expression may counteract ALS pathogenesis through multiple mechanisms, including reducing oxidative stress by scavenging reactive oxygen species (ROS), restoring mitochondrial homeostasis, suppressing stress granule dysregulation, inhibiting neuroinflammatory cascades, and enhancing neuronal membrane repair capacity.

清华大学实验室。SNUG01利用rAAV9载体通过鞘内注射将人类TRIM72基因递送到神经元。临床前研究表明，TRIM72表达可能通过多种机制对抗ALS的发病机制，包括通过清除活性氧（ROS）减少氧化应激、恢复线粒体稳态、抑制应激颗粒失调、阻断神经炎症级联反应，以及增强神经元膜修复能力。

This multi-dimensional action delays motor neuron degeneration in ALS..

这种多维行动延缓了ALS中运动神经元的退化。

A recently completed investigator-initiated trial (IIT) in China demonstrated SNUG01's favorable safety and tolerability profile, alongside early signs of clinical efficacy and biomarker improvement. Crucially, unlike ALS therapies targeting specific genetic mutations, SNUG01's broad neuroprotective mechanism holds the potential to benefit over 90% of ALS patients with sporadic disease – a population currently lacking effective treatment options.

最近在中国完成的一项研究者发起的试验 (IIT) 显示，SNUG01 具有良好的安全性和耐受性，并且有早期临床疗效和生物标志物改善的迹象。重要的是，与针对特定基因突变的 ALS 疗法不同，SNUG01 广泛的神经保护机制有可能使超过 90% 的散发型 ALS 患者受益——这一群体目前缺乏有效的治疗选择。

These promising results support its continued global development..

这些令人鼓舞的结果支持其继续在全球范围内发展。

About ALS

关于ALS

ALS is a rapidly progressive neurodegenerative disorder marked by motor neuron degeneration in the brain and spinal cord, leading to muscle weakness, atrophy, and eventually respiratory failure. Median survival time is 3–5 years. Despite being the most prevalent adult-onset motor neuron disease, existing therapies offer limited clinical benefits, slowing progression only marginally and leaving no curative options for patients..

ALS是一种快速进展的神经退行性疾病，其特征是大脑和脊髓中的运动神经元退化，导致肌肉无力、萎缩，并最终引发呼吸衰竭。中位生存时间为3至5年。尽管它是最常见的成人起病的运动神经元疾病，但现有的治疗方法临床效果有限，仅能略微延缓病情进展，且无法为患者提供治愈的选择。

About SineuGene

关于SineuGene

SineuGene Therapeutics Co., Ltd. ('SineuGene') is a clinical-stage biotech company pioneering innovative therapies, including AAV-based and nucleic acid-based treatments, for neurological disorders. Founded in late 2021, the company's initial pipeline was built upon foundational research led by Dr. .

SineuGene Therapeutics Co., Ltd.（“SineuGene”）是一家临床阶段的生物技术公司，致力于开发创新疗法，包括基于AAV和核酸的治疗方法，用于神经系统疾病。公司成立于2021年底，其初始研发管线基于Dr. 领导的基础研究。

Yichang Jia

宜昌贾

at Tsinghua University's School of Medicine. SineuGene leverages cutting-edge technologies, such as adeno-associated virus (AAV)-mediated gene delivery and antisense oligonucleotide (ASO)-based gene regulation, to develop a robust pipeline targeting central nervous system disorders including ALS, stroke, Parkinson's disease, Alzheimer's disease, Multiple System Atrophy, Spinocerebellar Ataxia type 3, and Huntington's disease.

在清华大学医学院。SineuGene利用尖端技术，如腺相关病毒（AAV）介导的基因递送和基于反义寡核苷酸（ASO）的基因调控，开发出一条强大的管线，针对中枢神经系统疾病，包括肌萎缩侧索硬化症（ALS）、中风、帕金森病、阿尔茨海默病、多系统萎缩、脊髓小脑共济失调3型和亨廷顿病。

SineuGene is committed to translating scientific discoveries into transformative therapies for patients with serious unmet medical needs..

SineuGene致力于将科学发现转化为满足严重未满足医疗需求患者的变革性疗法。