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SineuGene's Game-Changing Gene Therapy for ALS

On March 24, 2025, SineuGene Therapeutics Co., Ltd. announced an important milestone in the fight against Amyotrophic Lateral Sclerosis (ALS) with the FDA granting clearance for its Investigational New Drug (IND) application for SNUG01. Distinctively, SNUG01 is a first-in-class gene therapy that focuses on the TRIM72 protein, known for its neuroprotective properties. The clearance allows SineuGene to advance to a global Phase I/IIa clinical trial to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 for adults diagnosed with ALS.

ALS is a devastating progressive neurodegenerative condition that primarily affects motor neurons, leading to severe muscle weakness, atrophy, and eventual respiratory failure, often within just three to five years of diagnosis. Current treatments provide minimal benefits, highlighting the urgent need for new therapies that can significantly improve patient outcomes.

What sets SNUG01 apart is its foundation in pioneering research conducted by Dr. Yichang Jia at Tsinghua University. The research established TRIM72 as a multifunctional neuroprotectant, enabling SNUG01 to attack ALS from multiple angles. Using a recombinant adeno-associated virus serotype 9 (rAAV9), it aims to deliver the human TRIM72 gene directly into the central nervous system via intrathecal administration. Preclinical studies have demonstrated that TRIM72 helps combat the disease's progression by reducing oxidative stress, restoring mitochondrial function, managing stress granules, suppressing neuroinflammation, and enhancing neuronal repair mechanisms.

In early trials conducted at Peking University Third Hospital, SNUG01 showed a promising safety profile and preliminary signs of efficacy, with improvements noted in functional clinical assessments and neurodegeneration biomarkers. These positive indicators strengthen the therapy's potential in bridging the gap between groundbreaking research and human clinical applications, reinforcing its role as a frontrunner in ALS treatment.

Unlike conventional gene therapies that target specific mutations, SNUG01 utilizes a multifaceted approach, making it suitable for the over 90% of ALS patients diagnosed with sporadic forms of the disease. This flexibility positions SNUG01 as a crucial option for many who currently have few, if any, treatment options.

To expedite its clinical development, SineuGene intends to collaborate with an esteemed global consortium of academic and clinical institutions to conduct extensive multi-regional trials that will further validate the safety and therapeutic efficacy of SNUG01 across diverse populations of ALS patients.

About SineuGene

Established in late 2021, SineuGene Therapeutics is committed to developing innovative solutions for debilitating neurological disorders. This biotech firm builds upon over ten years of dedicated scientific research spearheaded by Dr. Yichang Jia and his team at Tsinghua University’s School of Medicine. By utilizing advanced methodologies such as AAV-based gene therapies and antisense oligonucleotide regulation, SineuGene is expanding its pipeline to target various neurological conditions like stroke, Parkinson's disease, Alzheimer's disease, Spinocerebellar Ataxia type 3, and Huntington's disease. Their mission focuses on transforming cutting-edge research into effective treatments to meet the critical medical needs in neurology.

For more information, visit SineuGene's website.