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AskBio Reports Safety Data from AB-1003 LGMD 2I/R9 Study

AskBio Reports Safety Data from AB-1003 LGMD 2I/R9 Study

AskBio has reported initial safety data from the first cohort of participants from its phase-I/phase-II LION-CS101 clinical trial of investigational gene therapy AB-1003 in participants with Limb-Girdle Muscular Dystrophy (LGMD) 2I/R9 at the 30th Annual International Congress of the World Muscle Society, that took place from October 7-11, 2025, in Vienna, Austria.

The presentation represented interim, blinded Cohort 1 safety data. Participants enrolled in Cohort 1 received a single intravenous infusion of AB-1003 or placebo and were followed for 52 weeks post-treatment during the main trial before entering a planned four-year long-term follow-up period. Safety assessments included Adverse Event (AE) monitoring, laboratory testing, physical exams, vital signs, electrocardiograms and echocardiograms.

There were no dose-limiting toxicities or serious AEs reported up to 52 weeks post-treatment. Commonly reported (>2 participants) treatment-emergent adverse events were mild-to-moderate in severity and included headaches, falls and nausea. Three participants reported asymptomatic transient transaminase elevations without changes in bilirubin levels, which returned to baseline levels after adjusting corticosteroid treatment.

The data was presented by Chris Passalacqua, MD, Vice President—Neuromuscular Medical Affairs, AskBio, on October 10, 2025.

“These initial safety data are encouraging and suggest an acceptable safety profile for AB-1003. We believe AAV-mediated gene therapy has the potential to restore FKRP function and stabilise disease progression, and we are excited to continue our clinical research efforts with the goal of developing an effective treatment for Limb-Girdle Muscular Dystrophy,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.

The LION-CS101 clinical trial is a double-blind, randomised, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9. The trial includes two sequential, dose-level cohorts. Adult participants diagnosed with LGMD2I/R9 will receive a single intravenous infusion of AB-1003 or placebo. The trial was initiated in 2023.

 

More news about: clinical trials | Published by Dineshwori | October - 13 - 2025

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