(Yicai) March 24 -- Chinese biotech startup SineuGene Therapeutics has gained US approval for clinical research on a potentially groundbreaking gene therapy for amyotrophic lateral sclerosis, a neurodegenerative disease that leads to muscle weakness and eventual paralysis.

Snug01, the world's first ALS treatment targeting the human Tripartite Motif Protein 72, received the investigational new drug approval from the US Food and Drug Administration on March 21,  Beijing-based SineuGene announced today, adding that it will shortly enter the international multicenter clinical trial phase.

The average survival time for ALS patients is only three to five years, and there are no cures available yet, so the focus is solely on therapies to slow the disease’s progression.

SineuGene developed Snug01 based on original discoveries at Jia Lab in Tsinghua University. The drug delivers the human Trim72 gene to the neurons of ALS patients through injection directly into the cerebrospinal fluid surrounding the brain and spinal cord. It is expected to be effective in various ways, including cell membrane repair, antioxidant effects, and mitochondrial function restoration, potentially slowing neuronal damage.

SineuGene began clinical research on Snug01 at Peking University Third Hospital, and so far it has shown good safety and tolerability as well as positive signals in efficacy markers, according to the announcement.

Founded in late 2021, SineuGene aims to develop treatments for neurological diseases such as ALS, stroke, Parkinson's disease, Alzheimer's disease, Huntington's chorea, and autism spectrum disorder, according to its website.

Editor: Tom Litting