Bayer arm advances Gene therapy clinical trial for Limb-Girdle Muscular Dystrophy Type 2I/R9

The trial was initiated in 2023. It will include up to 14 participants at six sites throughout the United States. Participants in the first cohort remain in the study until completion. Enrollment in cohort two is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com.

“The burden of LGMD2I/R9 on patients and their families is profound,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9. This is a rare and debilitating type of muscular dystrophy, and this advancement brings the LION-CS101 trial another step closer to completion.”

AskBio has received rare pediatric disease designation (RPDD), orphan-drug designation (ODD) and fast track designation (FTD) for AB-1003 for the potential treatment of LGMD2I/R9 from the United States Food and Drug Administration (FDA). These designations serve as clear recognition of the significant unmet medical need in LGMD2I/R9, for which there is no approved therapy.

“The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We are encouraged by the DSMB’s recommendation to advance our study, following their thorough assessment of AB-1003 in cohort one and are excited to proceed with the second cohort.”

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 FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion.