Askbio Commences Phase 1/phase 2 Clinical Study of AB-1003 Gene Therapy for Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
Friday, August 04, 2023
Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has recently announced the commencement of the Phase 1/Phase 2 LION-CS101 clinical trial for patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This clinical trial is focused on evaluating the safety and efficacy of AB-1003 (also known as LION-101), a novel investigational FKRP gene replacement therapy.
LGMD2I/R9 is a rare form of limb-girdle muscular dystrophy caused by mutations in the FKRP gene, resulting in muscle weakness and atrophy in the arms and legs. It is estimated to affect more than 5,000 people in the US and EU. The disease typically manifests in late childhood, around 11 years of age, and leads to the loss of mobility, as well as impaired heart and lung function, eventually necessitating the use of wheelchairs.
AB-1003 aims to introduce a normal FKRP gene into the muscle cells, potentially restoring normal protein function and alleviating the symptoms of LGMD2I/R9. Promising results have been observed in preclinical studies using mouse models of LGMD.
The LION-CS101 clinical trial is a Phase 1/Phase 2 study that will assess the safety and efficacy of AB-1003 in adult subjects (ages 18 to 65) with genetically confirmed LGMD2I/R9. The trial will be double-blind, randomized, placebo-controlled, and will involve sequential, dose-escalation treatment with AB-1003 or placebo. Up to 14 participants will be enrolled in the trial, which commenced in the US in Q2 2023.
AB-1003 has been granted fast track designation by the FDA and orphan drug designation by the European Commission, underscoring its potential as a treatment for LGMD2I/R9.
This significant development in dosing the first patient in the LION-CS101 trial represents a major step forward for AskBio and its subsidiary, Viralgen. It showcases their commitment to advancing gene therapies for various devastating diseases, including limb-girdle muscular dystrophy, congestive heart failure, Huntington's disease, multiple system atrophy, Parkinson's disease, and Pompe disease.
The initiation of this trial offers hope to individuals living with LGMD2I/R9, as it holds the promise of potentially delivering much-needed disease-modifying treatments. The recruitment for the LION-CS101 clinical trial is currently ongoing, and interested parties can find more information at clinicaltrials.gov (NCT05230459) or askbio.com.
