SineuGene’s ALS Gene Therapy SNUG01 Granted FDA Orphan Drug Designation

Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders, announced this week that its first-in-class (FIC) TRIM72-targeted gene therapy SNUG01 has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS).

Therapy Innovation
SNUG01 is the world’s first gene therapy targeting TRIM72 and was developed by SineuGene using its proprietary AAV platform technology. The target was identified through next-generation gene knock-in ALS models developed by Prof. Jia Yichang’s laboratory at Tsinghua University School of Basic Medical Sciences.

Mechanism and Delivery
Utilizing recombinant AAV9 (rAAV9) as the delivery vector, SNUG01 achieves neuron-specific TRIM72 gene delivery via intrathecal administration (IT). Preclinical studies suggest that TRIM72 offers multi-mechanistic neuroprotection through enhanced membrane repair, restored antioxidant/mitochondrial function, and reduced stress granule formation, thereby slowing motor neuron degeneration in ALS patients.

Clinical Promise
Initial data from the investigator-initiated trial (IIT) demonstrated favorable safety and tolerability profiles for SNUG01, with promising signals in both efficacy endpoints and biomarker improvements.

Broader Implications
Unlike mutation-specific ALS therapies, SNUG01’s multidimensional neuroprotective mechanisms may benefit a broader ALS population, particularly offering potential for sporadic ALS cases (90% of patients) who currently lack effective treatments.-Fineline Info & Tech