First-in-Class ALS AAV Gene Therapy, SNUG01, to Enter Global Clinical Trial

BEIJING – SineuGene Therapeutics, a clinical-stage biotechnology company, announced today that China’s National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for its groundbreaking gene therapy product, SNUG01, for amyotrophic lateral sclerosis (ALS).

This milestone follows a similar clearance granted by the U.S. Food and Drug Administration (FDA) in March 2025, paving the way for the initiation of a global Phase I/IIa multi-regional clinical trial (MRCT) in both China and the United States.

SNUG01 is a first-in-class, recombinant AAV9-based gene therapy designed to deliver the human TRIM72 protein to motor neurons in the central nervous system. Preclinical studies suggest that TRIM72’s broad neuroprotective mechanism can counteract ALS progression by reducing oxidative stress, enhancing neuronal repair, and inhibiting neuroinflammatory pathways.

The upcoming trial will evaluate the safety and potential efficacy of SNUG01 in adults with ALS. The study will be conducted at multiple leading academic centers in both countries, including the Healey & AMG Center for ALS at Massachusetts General Hospital in the U.S.

“The dual clearance accelerates and validates the strategy for the development of potentially transformative therapies for people with ALS worldwide,” said Professor Merit Cudkowicz, global principal investigator of the trial.

The company noted that a recently completed investigator-initiated trial in China already demonstrated SNUG01’s favorable safety profile and early signs of clinical efficacy. A key advantage of the therapy is its potential to benefit the sporadic ALS patient population, which makes up over 90% of all ALS cases and currently has no effective treatment options.

PackGene’s Role

PackGene is a proud partner in the development of SNUG01, providing comprehensive AAV vector services from process development to GMP production. These services aim to accelerate the market launch of SineuGene’s gene therapy product.