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

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

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 characterised by Dr Yichang Jia's group at Tsinghua University, upon delivery to the central nervous system (CNS), particularly motor neurons. The company says that 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 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."

ALS is a rapidly progressive neurodegenerative disorder marked by motor neuron degeneration in the brain and spinal cord, leading to muscle weakness, atrophy, and eventually respiratory failure.