Type I SMA: a new genes promising in preclinical

To overcome possible toxicity problems with Zolgensma gene therapy^®on the heart. For example. Meanwhile, the liver, a new gene therapy developed for the SMA of type I, EXG001-307, could present a better security profile. Similarly, It includes a copy of the gene SMN1 Functional associated with a specific promoter to neurons which will allow. Therefore, from this gene, the manufacture of SMN protein in neural cells.

Increased efficiency. Similarly. less toxicity in treated mice – Type i sma: new genes

Researchers have thus highlighted mouse models with SMA the effectiveness of EXG001-307, with an increase in lifespan, greater weight gain and restoration of motor function and muscle strength.
The higher the dose, the greater the efficiency. In particular, the mice that received this dose survived 191 days (median) with a maximum of 319 type i sma: new genes new days. As for those processed by the product SCAAV9-CB-HSMN (identical to Zolgensma®), they survived 32.5 days (median) type i sma: new genes with a maximum of 124 days, the highest dose which caused significant toxicity.

In addition. the level of expression of the protein SMN In the heart was much lower in mice treated by EXG001-307 than in those treated by the vector identical to Zolgensma. which limits the risks in terms of security. While the mortality rate was high among the mouse treated at medium. high dose by SCAAV9-CB-HSMN, no death linked to EXG001-307 occurred.

These favorable results must then be confirmed in the current I/II test.

Source
Efficacy. preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy
Song C, Wang Q, Zhu P et al.
Mol Ther Methods Clin Dev. 2025 Apr.