Beijing-based SineuGene Therapeutics, a developer of gene therapies for brain disorders, announced last week that it has received clearance from the US Food and Drug Administration (FDA) to conduct a clinical study for its lead candidate SNUG01. This best-in-class (BIC) TRIM72-targeted gene therapy will be evaluated for the treatment of amyotrophic lateral sclerosis (ALS).
Pre-Clinical Results and Mechanism
In pre-clinical animal studies, SNUG01 demonstrated significant neuroprotective effects on neurons. The therapy has multiple protective mechanisms on nerve cells, making it a potential treatment option for a broader range of ALS subtypes. Unlike gene therapies targeting ALS caused by specific gene mutations, SNUG01 offers a novel approach, particularly for the 90% of patients with sporadic ALS who currently have no specific treatment options.
Clinical Trial Progress
An investigator-initiated trial further confirmed the drug’s safety and tolerability, with encouraging positive signals in efficacy indicators and biomarkers. This progress positions SNUG01 as a promising candidate in the field of ALS treatment, where there remains significant unmet medical need.-Fineline Info & Tech
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