SineuGene Therapeutics Co., Ltd. is a clinical-stage biotech company pioneering innovative therapies, focusing on AAV-based and nucleic acid-based treatments, for neurological disorders. Founded in late 2021, the company’s initial pipeline was built upon foundational research led by Dr. Yichang Jia at Tsinghua University's School of Medicine. SineuGene leverages cutting-edge technologies, such as adeno-associated virus (AAV) mediated gene delivery and antisense oligonucleotide (ASO) based gene regulation, to develop a robust pipeline targeting central nervous system disorders including ALS, Stroke, Parkinson’s Disease, Alzheimer’s Disease, Multiple System Atrophy (MSA), and Spinocerebellar Ataxia Type 3 (SCA3). 

In 2025, SNUG01, SineuGene’s lead program for ALS, received IND clearance from both the FDA and NMPA, as well as Orphan Drug Designation (ODD) from the FDA. The company is actively advancing an international China–US multi-center clinical trial. SineuGene is committed to translating scientific discoveries into transformative therapies for patients worldwide with unmet and urgent medical needs.