Currently, the management of LGMD2I/R9 focuses on symptom relief, as there are no approved therapies available for this debilitating condition.

In recognition of the unmet medical need for LGMD2I/R9 treatment, AskBio has received several designations from the FDA for AB-1003, including rare pediatric disease designation, orphan-drug designation, and fast track designation.

The decision to initiate the second cohort was made by an independent Data Safety Monitoring Board (DSMB) after reviewing safety data from the first cohort of the trial.

The trial, which began in 2023, aims to enroll up to 14 participants across six sites in the United States, with current enrollment for the second cohort ongoing.

AskBio Inc. has made significant progress in its Phase 1/Phase 2 LION-CS101 clinical trial for the gene therapy AB-1003, successfully dosing the first participant in the second cohort.

Designed as a double-blind, randomized, placebo-controlled study, the LION-CS101 trial evaluates the safety of AB-1003 in adult participants aged 18 to 65 who have a genetic confirmation of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

Nicholas Johnson, MD, the Principal Investigator of the trial, highlighted the importance of this milestone for patients suffering from LGMD2I/R9.

LGMD2I/R9 is a rare genetic disorder caused by mutations in the FKRP gene, affecting fewer than 5,000 individuals in the U.S., leading to muscle weakness and eventual loss of mobility.

AB-1003 is an investigational recombinant adeno-associated virus (AAV)-based gene therapy designed to restore FKRP enzyme activity and is administered as a one-time intravenous infusion.