Sarepta announced it has recently signed an agreement with the Research Institute at Nationwide Children’s Hospital giving Sarepta the exclusive option to a Nationwide Children’s gene therapy candidate, calpain 3, or CAPN-3, to treat Limb-girdle muscular dystrophy type 2A, or LGMD2A. LGMDs represent a group of distinct genetic neuromuscular diseases with a generally common set of symptoms, including progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. Many LGMD sub-types are life-limiting and often life-ending diseases. Also known as calpainopathy, LGMD2A is caused by mutations in the CAPN-3 gene and is the most common type of LGMD, accounting for almost a third of cases. Like Sarepta’s micro-dystrophin and five other LGMD programs, the LGMD2A program employs the AAVrh74 vector, designed to deliver treatment to skeletal muscle, including the diaphragm, without crossing the blood brain barrier. The CAPN-3 program is currently in pre-clinical trials.
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