NaPro awarded grant for MD research

NaPro BioTherapeutics has received a small business innovation research (SBIR) award from the National Institute of Neurological Disorders and Stroke of the National Institutes of Health (NINDS/NIH) to use its gene editing technology to address inherited mutations in animal models of Duchenne muscular dystrophy (DMD).

NaPro will be targeting the existing mutant DMD gene with specifically designed proprietary oligonucleotides, which pair with, and direct, a muscle cell's natural repair mechanism to correct the inherited chromosomal defect. Initial studies will focus on dystrophinopathies in muscle tissue of murine and canine models of DMD.

Dr Frank Tagliaferri, director of Preclinical Development at NaPro and principal investigator for this work commented: 'NIH funding of NaPro in this area of research is enormously appreciated and represents an added source of recognition for our gene editing technology. This grant allows us to expand the therapeutic application of our technology to include not only hematologic and neurologic disorders, which we are currently working on, but also to include inherited mutations in muscle disorders as well.'

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is caused by a mutation on the X-chromosome whereby the protein dystrophin cannot be produced. This protein is necessary for normal muscle function to occur. The absence of dystrophin leads to progressive muscle wasting and weakness in the arms, legs, and trunk. The heart and respiratory muscles may also be affected. This is a hereditary disease affecting almost exclusively males and is usually fatal by 20-24 years of age. Approximately 1 in 3,300 males suffers from this disease in the United States.