SineuGene has received clearance from China's National Medical Products Administration (NMPA) to begin clinical trials for SNUG01, its TRIM72-targeted gene therapy for amyotrophic lateral sclerosis (ALS). The recombinant AAV9 vector delivers human TRIM72 via intrathecal injection, showing pre-clinical neuroprotection through membrane repair and mitochondrial function modulation. Early investigator-led trials demonstrated safety and biomarker improvements, with potential applicability to 90% of sporadic ALS cases lacking targeted treatments.

The approval enables SineuGene to proceed with global Phase I/II trials following receipt of orphan drug designation (ODD) from the US in June 2025. Developed from Tsinghua University research, SNUG01 aims to address ALS's 3-to-5-year median survival by targeting multiple degenerative pathways. The company plans multinational studies to evaluate safety and efficacy in slowing motor neuron degeneration.

According to PharmCube's NextBiopharm® database, a total of 62 gene therapies are currently under active development in ALS globally. Click here to request a free trial for NextBiopharm®.

Source:
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