NEW YORK – Asklepios BioPharmaceutical on Thursday said the first patient with limb-girdle muscular dystrophy type 2I/R9 has received its investigational gene therapy AB-1003 in a Phase I/II trial.
The Bayer subsidiary, called AskBio for short, has designed AB-1003 to replace the mutated form of the FKRP gene with a normal version. Mutations in FKRP cause limb-girdle muscular dystrophy type 2I/R9, a disorder in which arm and leg muscles progressively weaken. By replacing the faulty FKRP gene, the drug is designed to restore normal fukutin-related protein activity in muscle cells.
In the double-blind, randomized, placebo-controlled Phase I/II trial, dubbed LION-CS101, AskBio will test the safety of escalating doses of its gene therapy in adults with genetic confirmation of the rare muscular dystrophy disorder. Investigators plan to enroll up to 14 participants.
"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," Nicholas Johnson, a principal investigator and vice chair of research in the neurology department at Virginia Commonwealth University School of Medicine, said in a statement.
AB-1003, also known as LION-101, is manufactured by Viralgen, a subsidiary of AskBio and an independently operated contract development and manufacturing organization.