SineuGene's SNUG01 ALS Gene Therapy Gets FDA IND Clearance

BEIJING, China, March 24, 2025 - SineuGene Therapeutics Co., Ltd. has reached a significant milestone in its mission to develop therapies for neurological disorders. The company announced that the U.S. Food and Drug Administration (FDA) approved its Investigational New Drug (IND) application for SNUG01, a pioneering gene therapy candidate specifically targeting TRIM72 (Tripartite Motif Protein 72) for the treatment of amyotrophic lateral sclerosis (ALS). This approval paves the way for a global Phase I/IIa clinical trial, which will explore the safety, tolerability, and initial effectiveness of SNUG01 in adults diagnosed with ALS through a study involving dose-escalation and expansion.

ALS is a rapidly advancing neurodegenerative disease that results in the degeneration of motor neurons in the brain and spinal cord. This degeneration leads to muscle weakness, atrophy, and eventually respiratory failure, with most patients having a median survival of three to five years after diagnosis. It is the most common adult-onset motor neuron disease, yet current treatments provide minimal slowdown in progression and no cure, leaving a substantial treatment gap.

The development of SNUG01 is built on innovative research from Dr. Yichang Jia’s lab at Tsinghua University, which identified TRIM72 as a key multifunctional neuroprotective agent. The therapy employs a recombinant adeno-associated virus serotype 9 (rAAV9) to deliver the human TRIM72 gene directly into the spinal fluid. Preclinical studies have shown that TRIM72 can counteract ALS by employing multiple mechanisms. These include reducing oxidative stress by scavenging reactive oxygen species, restoring mitochondrial function, preventing stress granule irregularities, hindering neuroinflammation, and enhancing the repair capacity of neuronal membranes.

In an early-stage trial conducted at Peking University Third Hospital, SNUG01 exhibited a positive safety and tolerability profile, along with promising efficacy signals as evidenced by improvements in functional assessments and biomarkers associated with neurodegeneration. The findings provide strong support for the translation of TRIM72 from a scientific discovery to a therapeutic concept proven in humans.

The therapy stands out due to its broad application, as it does not solely target genetic mutations. Instead, SNUG01 leverages multiple neuroprotective pathways, offering substantial benefits for over 90% of ALS patients who suffer from sporadic, non-familial forms of the disease. This positions SNUG01 as a unique therapeutic candidate, potentially fulfilling a significant unmet need among a vast majority of ALS patients.

SineuGene plans to collaborate with a distinguished network of academic and clinical research institutions globally to conduct a multi-regional clinical trial. This trial aims to accelerate the validation of SNUG01’s safety and therapeutic efficacy across diverse ALS patient groups.

SineuGene Therapeutics Co., Ltd., established in late 2021, is committed to advancing neurological disease treatment through innovation. The company’s foundation lies in over ten years of scientific inquiry led by Dr. Yichang Jia at the School of Medicine, Tsinghua University. SineuGene employs cutting-edge technologies, including adeno-associated virus (AAV) vector-mediated gene delivery and editing, as well as antisense oligonucleotide (ASO) for gene expression regulation. Its diverse research pipeline targets major neurological disorders such as ALS, stroke, Parkinson’s disease, Alzheimer’s disease, Spinocerebellar Ataxia type 3, and Huntington’s disease, striving to transform pioneering research into viable therapies that address critical medical needs in neurology.