SineuGene Therapeutics Receives Clearance on SNUG01 for Gene Therapy Targeting Amyotrophic Lateral Sclerosis (ALS) Treatment
Tuesday, March 25, 2025
SineuGene Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for SNUG01, a gene therapy candidate targeting TRIM72 (Tripartite Motif Protein 72) for the treatment of amyotrophic lateral sclerosis (ALS).
ALS is a progressive neurodegenerative condition affecting motor neurons in the brain and spinal cord. The disease leads to muscle weakness, atrophy, and eventual respiratory failure. The median survival period following diagnosis is typically between three and five years.
Current treatments provide limited benefits, offering only a slight reduction in disease progression without curative options.
SNUG01 is based on research from Tsinghua University, where TRIM72 was identified as a multifunctional neuroprotective factor. The therapy employs a recombinant adeno-associated virus serotype 9 (rAAV9) to deliver the human TRIM72 gene via intrathecal administration.
Preclinical studies suggest that TRIM72 mitigates ALS progression through several mechanisms, including reducing oxidative stress, restoring mitochondrial function, regulating stress granules, inhibiting neuroinflammation, and enhancing neuronal membrane repair.
The approval allows the initiation of a global Phase I/IIa clinical trial to assess the safety, tolerability, and initial effectiveness of SNUG01 in adults with ALS through a dose-escalation and expansion study.
Early results also showed promising signs of efficacy based on clinical assessments and key biomarkers associated with neurodegeneration. These findings support the transition of TRIM72 research from discovery through preclinical models to initial human studies.
Unlike therapies targeting specific genetic mutations, SNUG01 operates through multiple neuroprotective pathways, making it suitable for more than 90 per cent of ALS cases, which occur sporadically rather than through inherited mutations.
The study aims to further evaluate SNUG01’s safety and therapeutic potential across diverse patient populations, supporting the development of new treatment options for ALS.
This broad applicability offers a potential treatment option for a significant proportion of ALS patients.
SineuGene plans to collaborate with international academic and clinical institutions to conduct a multi-regional clinical trial.
Source: prnewswire.com
