Photo of PUTH and SineuGene Team.

“These initial findings are encouraging,” said Professor Fan Dongsheng. “SNUG01 has shown an promising safety profile and early signals of efficacy, which warrant further investigation in larger studies.”

Dr. Jia Yichang, founder of SineuGene and professor of Tsinghua University, added: “The results from this trial reinforce the potential of SNUG01 as a new treatment for ALS. We are deeply grateful to Professor Fan and his team at PUTH for their invaluable contributions to this study. We also want to express our heartfelt thanks to the patients and their families for their trust and participation. Their support has been critical to advancing this important research.”

About SNUG01

SNUG01 is an investigational gene therapy drug that uses adeno-associated virus (AAV) vectors to deliver SG001, a molecule designed to protect motor neurons through various neuroprotective mechanisms. Preclinical studies demonstrated its ability to protect motor neurons, making it a promising approach for ALS treatment.

About ALS

ALS is a progressive neurodegenerative disease that affects motor neurons in the brain and spinal cord, leading to muscle weakness, loss of function, and eventual death. The median survival after diagnosis is 2-5 years, with limited treatment options available.

About SineuGene

SineuGene Therapeutics is a biotechnology company specializing in gene therapies for neurological disorders. Founded in late 2021, the company builds on over a decade of neuroscience research from JiaLab at Tsinghua University’s School of Medicine. SineuGene has developed cutting-edge platforms for advanced animal models, adeno-associated virus (AAV) screening, and small nucleic acid therapies. Leveraging its expertise in gene expression and editing, SineuGene is focused on developing treatments for ALS, stroke, Parkinson’s, Alzheimer’s, and other neurodegenerative diseases.