Novel Gene Therapy for ALS cleared for global trials in China and the U.S.

BEIJING, Aug. 15, 2025 /PRNewswire/ -- SineuGene Therapeutics Co., Ltd. ("SineuGene"), clinical-stage biotechnology company that mainly focuses on gene therapies for neurological disorders, announced today that China's National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for SNUG01, a groundbreaking gene therapy product for amyotrophic lateral sclerosis (ALS). This milestone follows a prior clearance of SNUG01's IND application by the U.S. Food and Drug Administration (FDA) in March 2025, paving the way for the initiation of a Phase I/IIa multi-regional clinical trial (MRCT) in both China and the United States.

SNUG01 is a first-in-class recombinant adeno-associated virus serotype 9 (rAAV9) based gene therapy product that expresses the human TRIM72 protein (Tripartite Motif Protein 72), a new drug target for ALS initially identified and characterized by Dr. Yichang Jia's group at Tsinghua University, upon delivery to the central nervous system (CNS), particularly motor neurons. This global Phase I/IIa trial will evaluate the safety and potential efficacy of SNUG01 and identify dosage for further clinical development in adults with ALS. The study will be conducted in multiple leading academic centers in both countries, including the Healey & AMG Center for ALS at Massachusetts General Hospital (U.S.), Peking University Third Hospital, the Second Affiliated Hospital of Zhejiang University School of Medicine and Fujian Medical University Union Hospital.

"The dual clearance accelerates and validates the strategy for the development of potentially transformative therapies for people with ALS worldwide", said Professor Merit Cudkowicz, Director of the Healey & AMG Center for ALS at Massachusetts General Hospital and global principal investigator of the trial. "We are pleased to collaborate on this study and look forward to evaluating the therapy's potential impact on patients living with ALS."

The regulatory clearances that have been conducted in both the U.S. and China, demonstrating the momentum behind SNUG01's global development. Through strategic collaborations and scientific rigor, SineuGene is advancing its first-in-class gene therapy to address the unmet and urgent needs of the global ALS community.

About 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. TRIM72 was identified as a potential therapeutic gene based on foundational research from Dr. Yichang Jia's lab at Tsinghua University. SNUG01 utilizes a rAAV9 vector to deliver the human TRIM72 to neurons via intrathecal administration. Preclinical studies have shown that TRIM72 expression could counteract ALS pathogenesis through multiple mechanisms, including reducing oxidative stress, enhancing neuronal membrane repair capacity, restoring mitochondrial homeostasis, inhibiting neuroinflammatory cascades, and suppressing stress granule dysregulation. This multi-dimensional action delays motor neuron degeneration in patients with ALS.

A recently completed investigator-initiated trial (IIT) in China demonstrated SNUG01's favorable safety, along with early signs of clinical efficacy and biomarker improvements. Crucially, unlike ALS therapies targeting specific genetic mutations, SNUG01's broad neuroprotective mechanism holds the potential to benefit the sporadic ALS patients —a population occupying over 90% of ALS patients and currently lacking effective treatment options. These promising results support SNUG01's continued global development.

About 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 marginally and leaving no curative options for patients.

About SineuGene

SineuGene Therapeutics Co., Ltd. is a clinical-stage biotech company pioneering innovative therapies, focusing on 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. Yichang Jia 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 (MSA), and Spinocerebellar Ataxia Type 3 (SCA3). SineuGene is committed to translating scientific discoveries into transformative therapies for patients worldwide with unmet and urgent medical needs.

Website: www.sineugene.com  
Contact:
Ms YAN, Siying
[email protected] 

SOURCE SineuGene Therapeutics