October 10, 2025

Research Triangle Park, N.C. – October 10, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced it will present initial safety data from the first cohort of participants from its Phase 1/Phase 2 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, taking place October 7–11, 2025, in Vienna, Austria.  The presentation represents 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 monitoring, laboratory testing, physical exams, vital signs, electrocardiograms, and echocardiograms.1 There were no dose-limiting toxicities or serious adverse events 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.1 The data will be presented by Chris Passalacqua, MD, Vice President of Neuromuscular Medical Affairs, AskBio, at 3:45 p.m. CEST Friday, October 10, 2025. “These initial safety data are encouraging and suggest an acceptable safety profile for AB-1003,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We believe AAV-mediated gene therapy has the potential to restore FKRP function and stabilize 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.” The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation 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. It will include up to 14 participants at six sites throughout the United States. Five participants were enrolled in the first cohort. All are actively participating and should remain in the trial until completion. Enrollment in Cohort 2 is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com. AB-1003 is an investigational recombinant adeno-associative virus (AAV)-based gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. It is designed to restore fukutin-related protein (FKRP) enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion.2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused by mutations in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.5 Symptoms may start to appear from childhood to adulthood, and affected individuals may experience difficulty running and walking. The symptoms gradually worsen over time, and ...read the news