Oligonucleotide Opportunities in DMD with PepGen's James McArthur, Ph.D.

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Among the more prevalent genetic conditions, Duchenne muscular dystrophy  affects an estimated one in 3,500 male births worldwide.  It's caused by mutations of the DMD gene, which  regulates the production of a protein called dystrophin. Approved DMD treatments haven't demonstrated strong clinical outcomes, but James McArthur, Ph.D. and his team at PepGen are seeking to change that with a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates derived from the company's Enhanced Delivery Oligonucleotide platform. The Business of Biotech caught up with Dr. McArthur at PepGen's Cambridge headquarters to learn more. 

Catch up on more gene therapy development advances on bioprocessonline.com.

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