By Fineline Cube 
Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders based in Beijing, announced that it has received clearance from China’s National Medical Products Administration (NMPA) to conduct a clinical study for its first-in-class (FIC) gene therapy, SNUG01, for the treatment of amyotrophic lateral sclerosis (ALS). This marks a significant milestone in the development of innovative therapies for ALS.
SNUG01 Mechanism
SNUG01, developed using SineuGene’s proprietary AAV platform technology, is the world’s first gene therapy targeting TRIM72. The therapy utilizes recombinant AAV9 (rAAV9) as the delivery vector and is administered via intrathecal (IT) injection to achieve neuron-specific TRIM72 gene delivery.
Preclinical Findings
Preclinical studies have shown that TRIM72 offers multi-mechanistic neuroprotection through enhanced membrane repair, restored antioxidant and mitochondrial function, and reduced stress granule formation. These mechanisms help slow motor neuron degeneration in ALS patients.
Clinical Trial Progress
Initial data from the investigator-initiated trial (IIT) demonstrated that SNUG01 has favorable safety and tolerability profiles. Promising signals were observed in both efficacy endpoints and biomarker improvements, highlighting its potential as a novel treatment option for ALS patients.-Fineline Info & Tech