August 3, 2023

Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9. Research Triangle Park, N.C.– August 3, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been dosed in the Phase 1 / Phase 2 LION-CS101 clinical trial of patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).[1] AB-1003 (also known as LION-101) is a novel investigational FKRP gene replacement therapy. “Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease,” said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i. “Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy. The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life.” LGMD2I/R9 is a form of LGMD caused by changes in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.[2]Those with LGMD2I/R9 notice symptoms in late childhood, around 11 years of age, which may include loss of mobility and impaired heart and lung function.2,[3] As symptoms worsen, individuals generally require wheelchairs.2 LGMD2I/R9 is a rare disease, estimated to affect more than 5,000 people in the US and EU.[4] “While the inherited nature of limb-girdle muscular dystrophy means those with the FKRP gene mutation can’t produce a normal FKRP protein for physiological muscle function, AB-1003 is designed to introduce the normal FKRP gene into the muscle and express a normal protein, and it has shown promise in restoring normal FKRP protein function in muscle in preclinical studies performed in mouse models of LGMD,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “This trial is the first step toward evaluating the safety of AB-1003 and assessing the potential that AB-1003 has to improve the lives of patients with this serious, inherited ultra rare condition.” “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is another example of AskBio’s success in taking gene therapy candidates, such as AB-1003, from the bench into clinical development,” said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer of AskBio. “With our knowledge and expertise, we are working to harness the power of gene therapies as potential treatments for many of the world’s most devastating diseases. Our investigational therapy for limb-girdle muscular dystrophy is one of these and an important part of our portfolio, which also includes clinical stage investigational therapies for congestive heart failure, Huntington’s disease, multiple system atrophy, Parkinson’s disease, and Pompe disease.” AB-1003, which is manufactured by AskBio’s wholly owned and independently operated subsidiary Viralgen, was granted fast track designation by the FDA and orphan drug designation by the European Commission.[5],[6] Recruitment for the LION-CS101 clinical ...read the news