Bayer subsidiary Asklepios BioPharmaceutical (AskBio) has dosed the first patient in the Phase I/II LION-CS101 study of AB-1003 gene replacement therapy to treat limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).

Initiated in the US in the second quarter of this year, the dose-escalation, placebo-controlled, randomised, double-blind trial aims to assess the safety of AB-1003 in adult subjects.

Approximately 14 adults aged 18 to 65 years with genetic confirmation of LGMD2I/R9 have been enrolled.

Participants will receive treatment in sequential, dose-level cohorts, with two arms of different doses of AB-1003 and one placebo arm. The dosages will be given intravenously.

Asklepios BioPharmaceutical co-founder and chief scientific officer Jude Samulski said: “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.

“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.

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“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.”

The rare disease LGMD2I/R9 is associated with weakness and wasting of the arm and leg muscles.

The gene therapy introduces normal FKRP genes into the muscle, thereby restoring protein function in the muscle.

Manufactured by AskBio’s wholly owned subsidiary Viralgen, AB-1003 has previously received orphan drug designation from the European Commission and fast track designation from the FDA.

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