AskBio Reports Positive Interim Safety Data from LION-CS101 Gene Therapy Trial for LGMD2I/R9

AskBio has shared early safety results from the first cohort of its ongoing Phase I/II LION-CS101 trial assessing AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9. Presented at the 30th Annual International Congress of the World Muscle Society in Vienna, the interim analysis included adults aged 18–65 years who received a single intravenous dose of AB-1003 or placebo. Participants were monitored for 52 weeks before entering a planned long-term follow-up phase.

No dose-limiting toxicities or serious adverse events were reported. Common treatment-emergent adverse events—such as headache, nausea, and falls—were mild to moderate. Three individuals experienced transient, asymptomatic elevations in liver enzymes that normalized after corticosteroid adjustments. These findings indicate that AB-1003 demonstrates an acceptable safety profile. The study, launched in 2023, plans to enrol 14 participants across six US sites, with recruitment for Cohort 2 underway.

13-10-2025