An optimised version of gene therapy being studied in SMA

Beijing researchers report the results of preclinical studies on a new-generation gene therapy for SMA:

• the new transgene (AAV9-coSMN1) contains an optimised codonVoir code génétique., carried by an adeno-associated virusCe sont les plus petits micro-organismes vivants. Pour vivre et se multiplier, ils parasitent des cellules dont ils détournent à leur profit la machinerie. of the AAV9 type,
• mice from the Taiwanese model and non-human SMA primates were used and studied after intrathecal injection of the transgene,
• this modification in the composition of the transgene led to an increase in the production of the SMN1 protein in vitro« Dans le verre », expériences réalisées en laboratoire en dehors de l’organisme. and an improvement in the functional and histological parameters of the motor neurons, with no reported tolerance problems.

Clinical studies are already underway with the same product developed and manufactured in China (OptimumGene®).

Preclinical evaluation of AAV9-coSMN1 gene therapy for spinal muscular atrophy: efficacy and safety in mouse models and non-human primates. Ma W, Wu Z, Zhao T et al. Mol Med. 2025 Apr.