/PRNewswire/ -- SineuGene Therapeutics Co., Ltd. ('SineuGene'), a clinical-stage biotech company pioneering innovative therapies for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SNUG01 — a first-in-class TRIM72 (Tripartite Motif Protein 72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS).

/PRNewswire/ -- 临床阶段生物技术公司SineuGene Therapeutics Co., Ltd.（简称"SineuGene"），致力于开发神经疾病创新疗法，今日宣布美国食品药品监督管理局（FDA）已批准其针对肌萎缩侧索硬化症（ALS）的首创新药SNUG01（靶向TRIM72（三部分基序蛋白72）的基因疗法候选药物）的研究性新药（IND）申请。

The clearance authorizes a global Phase I/IIa clinical trial designed to evaluate SNUG01's safety, tolerability, and preliminary efficacy in adults with ALS through a dose-escalation and expansion study..

该批准授权进行一项全球 I/IIa 期临床试验，旨在通过剂量递增和扩展研究评估 SNUG01 在成人 ALS 患者中的安全性、耐受性和初步疗效。

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 post-diagnosis 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..

肌萎缩侧索硬化症（ALS）是一种快速进展的神经退行性疾病，以大脑和脊髓中的运动神经元退化为特征，导致肌肉无力、萎缩，并最终引发呼吸衰竭。诊断后的中位生存时间为3至5年。尽管它是最常见的成人起病运动神经元疾病，现有疗法的临床益处有限，仅能略微延缓疾病进展，且尚无治愈方法可供患者选择。

SNUG01 leverages groundbreaking research carried out by Dr.

SNUG01利用了博士进行的开创性研究。

laboratory at Tsinghua University, whose team identified TRIM72 as a multifunctional neuroprotectant. The therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver the human TRIM72 gene via intrathecal administration. Preclinical studies demonstrate TRIM72's ability to counteract ALS pathogenesis through several synergistic mechanisms such as 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..

清华大学实验室的团队确定了TRIM72作为一种多功能神经保护剂。该疗法利用重组腺相关病毒血清型9（rAAV9）衣壳通过鞘内注射递送人类TRIM72基因。临床前研究表明，TRIM72能够通过多种协同机制来对抗ALS的病理发生，例如通过清除活性氧（ROS）减少氧化应激、恢复线粒体稳态、抑制应激颗粒失调、阻止神经炎症级联反应以及增强神经元膜修复能力。

In an investigator-initiated trial (IIT) at Peking University Third Hospital, SNUG01 demonstrated a favorable safety and tolerability profile, along with early efficacy signals in both functional clinical assessments and key biomarkers of neurodegeneration. These results enhance the translational validation of TRIM72, bridging target discovery, preclinical models, and human proof-of-concept..

在北京大学第三医院开展的一项研究者发起的试验 (IIT) 中，SNUG01 展现了良好的安全性和耐受性，并在功能临床评估和神经退行性病变的关键生物标志物方面显示出初步疗效信号。这些结果增强了 TRIM72 的转化验证，连接了靶点发现、临床前模型和人体概念验证。

Unlike mutation-targeting gene therapies, SNUG01 leverages multiple neuroprotective mechanisms, offering a critical advantage for over 90% of ALS patients with sporadic (non-familial) disease. This unique multi-target approach positions SNUG01 as a first-in-class candidate for the majority of ALS cases, addressing an urgent unmet need in this underserved population..

与靶向突变的基因疗法不同，SNUG01利用多种神经保护机制，为超过90%的散发型（非家族性）ALS患者提供了关键优势。这种独特的多靶点方法使SNUG01成为大多数ALS病例中的首例候选药物，满足了这一未被充分服务人群的迫切未满足需求。

SineuGene will partner with a leading global consortium of academic and clinical institutions to conduct a multi-regional clinical trial, expediting validation of SNUG01's safety and therapeutic efficacy profile across a diverse ALS patient populations.

SineuGene将与一个由学术和临床机构组成的全球领先联盟合作，开展多区域临床试验，加快验证SNUG01在多样化ALS患者群体中的安全性和治疗效果。

About SineuGene

关于SineuGene

SineuGene Therapeutics Co., Ltd., often referred to as 'SineuGene,' is a biotechnology company focused on developing innovative therapies for neurological diseases. Established in late 2021, the company builds on over a decade of scientific research conducted by Dr.

SineuGene Therapeutics Co., Ltd.，常被称为“SineuGene”，是一家专注于开发神经系统疾病创新疗法的生物技术公司。公司成立于2021年底，基于Dr.超过十年的科学研究成果而创立。

SineuGene uses advanced technologies such as adeno-associated virus (AAV) vector-mediated gene delivery and editing and antisense oligonucleotide (ASO)- based gene expression regulation. The company has created a diverse pipeline that targets major neurological diseases, including Amyotrophic Lateral Sclerosis (ALS), stroke, Parkinson's disease, Alzheimer's disease, Spinocerebellar Ataxia type 3, and .

SineuGene使用先进的技术，例如腺相关病毒（AAV）载体介导的基因递送与编辑以及基于反义寡核苷酸（ASO）的基因表达调控。该公司已建立了一个多样化的研发管线，针对主要的神经系统疾病，包括肌萎缩侧索硬化症（ALS）、中风、帕金森病、阿尔茨海默病、脊髓小脑共济失调3型等。

Huntington's disease. SineuGene is dedicated to transforming groundbreaking research into effective treatments, aiming to meet the unmet medical needs in the field of neurology.

亨廷顿病疾病。SineuGene致力于将开创性的研究转化为有效的治疗方法，旨在满足神经学领域未被满足的医疗需求。