SineuGene gets FDA clearance to advance ALS gene therapy into clinic

China-based biotech SineuGene Therapeutics announced that the U.S. FDA cleared its investigational new drug (IND) application for a potential first-in-class tripartite motif protein 72-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS).

The clearance authorizes the launch of a global phase 1/2a clinical trial designed to evaluate the safety, tolerability, and preliminary efficacy of the therapy, SNUG01, in adults with ALS through a dose-escalation and expansion study.

SineuGene’s therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver the human TRIM72 gene via intrathecal administration. Preclinical studies demonstrate TRIM72’s ability to counteract ALS pathogenesis through several synergistic mechanisms such as reducing oxidative stress by scavenging reactive oxygen species, restoring mitochondrial homeostasis, suppressing stress granule dysregulation, inhibiting neuroinflammatory cascades, and enhancing neuronal membrane repair capacity.

According to SineuGene, unlike mutation-targeting gene therapies, SNUG01 leverages multiple neuroprotective mechanisms, offering a critical advantage for over 90% of ALS patients with sporadic (non-familial) disease. This unique multi-target approach positions SNUG01 as a first-in-class candidate for the majority of ALS cases, addressing an urgent unmet need in the underserved population.