SineuGene’s SNUG01 ALS gene therapy cleared for global trials in China, U.S.

CHINA—SineuGene Therapeutics Co., Ltd., a clinical-stage biotechnology company specializing in gene therapies for neurological disorders, has announced a significant regulatory milestone.

China’s National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for SNUG01, an innovative gene therapy designed to treat amyotrophic lateral sclerosis (ALS).

This approval follows a similar green light from the U.S. Food and Drug Administration (FDA) in March 2025, marking a major step towards launching a Phase I/IIa multi-regional clinical trial (MRCT) across China and the United States.

SNUG01 is a pioneering gene therapy that uses a recombinant adeno-associated virus serotype 9 (rAAV9) to deliver the human TRIM72 protein to the central nervous system, particularly targeting motor neurons.

TRIM72 is a newly identified drug target for ALS discovered and studied by Dr. Yichang Jia and his team at Tsinghua University.

By introducing TRIM72, the therapy aims to address the underlying causes of ALS, a devastating neurological disease that currently has limited treatment options.

The upcoming global trial will primarily assess the safety and potential effectiveness of SNUG01 in adults diagnosed with ALS.

Additionally, the study will help determine the appropriate dosage to guide further clinical development.

This critical research will be conducted at prominent academic medical centers in both countries.

Notable sites include the Healey & AMG Center for ALS at Massachusetts General Hospital in the U.S., as well as Peking University Third Hospital, the Second Affiliated Hospital of Zhejiang University School of Medicine, and Fujian Medical University Union Hospital in China.

Professor Merit Cudkowicz, director of the Healey & AMG Center for ALS and the global principal investigator for the trial, emphasized the importance of the dual regulatory clearances.

He highlighted how these approvals not only accelerate the development process but also validate the therapeutic strategy behind SNUG01.

Professor Cudkowicz expressed enthusiasm for the collaboration and anticipation for seeing how this novel therapy could benefit patients living with ALS worldwide.

The approvals from both the U.S. and Chinese regulatory authorities underscore the growing momentum behind SNUG01’s global development.

SineuGene is leveraging strategic partnerships and maintaining scientific rigor to advance this first-in-class gene therapy.

Their goal is to meet the urgent, unmet needs of the global ALS community by providing new, transformative treatment options.

The progress with SNUG01 represents a hopeful advance in the fight against ALS, demonstrating the potential of gene therapies to change the landscape of neurological disease treatment not only in China and the U.S. but across the globe.

 With this multi-regional trial underway, there is renewed optimism for patients and families affected by ALS as research moves closer to delivering impactful new therapies.

Sign up HERE to receive our email newsletters with the latest news and insights from Africa and beyond. Also, follow us on our WhatsApp channel for updates.