We are pleased to share the publication of a long-term preclinical study on AAV9-mediated SMN1 gene therapy for spinal muscular atrophy (SMA), led by members of our scientific team.
The study, titled “Long-Term Safety and Efficacy of AAV9 Vectors Expressing Human SMN1 Gene: A Preclinical Study,” was published in Basic & Clinical Pharmacology & Toxicology (BCPT).
In this study, researchers evaluated the long-term safety and transgene expression of an AAV9 vector carrying a codon-optimized human SMN1 gene following a single intravenous administration in neonatal mice. Animals were followed for 24 weeks, with assessments including clinical observations, laboratory testing, histopathology, and transgene expression across multiple tissues.
The study demonstrated sustained and widespread SMN1 expression in key tissues, including the central nervous system, skeletal muscle, heart, and liver. Treated animals showed 100% survival during the study period, with no significant treatment-related abnormalities identified through clinical, laboratory, or histopathological assessments.
These findings provide important long-term preclinical evidence supporting the safety and translational potential of systemic AAV9-mediated SMN1 gene delivery and contribute to the continued development of gene therapy approaches for SMA.