SineuGene Therapeutics Co., Ltd. ("SineuGene"), clinical-stage biotechnology company that mainly focuses on gene therapies for neurological disorders, announced today that China's National Medical Products Administration (NMPA) has cleared the Investigational New Drug (IND) application for SNUG01, a groundbreaking gene therapy product for amyotrophic lateral sclerosis (ALS).

This milestone follows a prior clearance of SNUG01's IND application by the U.S. Food and Drug Administration (FDA) in March 2025, paving the way for the initiation of a Phase I/IIa multi-regional clinical trial (MRCT) in both China and the United States.

SNUG01 is a first-in-class recombinant adeno-associated virus serotype 9 (rAAV9) based gene therapy product that expresses the human TRIM72 protein (Tripartite Motif Protein 72), a new drug target for ALS initially identified and characterized by Dr. Yichang Jia's group at Tsinghua University, upon delivery to the central nervous system (CNS), particularly motor neurons. This global Phase I/IIa trial will evaluate the safety and potential efficacy of SNUG01 and identify dosage for further clinical development in adults with ALS. The study will be conducted in multiple leading academic centers in both countries, including the Healey & AMG Center for ALS at Massachusetts General Hospital (U.S.), Peking University Third Hospital, the Second Affiliated Hospital of Zhejiang University School of Medicine and Fujian Medical University Union Hospital.

"The dual clearance accelerates and validates the strategy for the development of potentially transformative therapies for people with ALS worldwide", said Professor Merit Cudkowicz, Director of the Healey & AMG Center for ALS at Massachusetts General Hospital and global principal investigator of the trial. "We are pleased to collaborate on this study and look forward to evaluating the therapy's potential impact on patients living with ALS."

The regulatory clearances that have been conducted in both the U.S. and China, demonstrating the momentum behind SNUG01's global development. Through strategic collaborations and scientific rigor, SineuGene is advancing its first-in-class gene therapy to address the unmet and urgent needs of the global ALS community.