CLINICAL TRIALS ARTICLES

CLINICAL TRIALS VIDEOS

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.

Dr. Francois Vigneault, Co-Founder, President, and CEO at Shape Therapeutics joins Cell & Gene: The Podcast's Erin Harris to discuss how RNA technologies are shaping the future of gene therapy. They also discuss AI's role in gene therapy as well as how the CGT sector is benefiting from RNA, AI, and synthetic biology.

Watch as experts in a multidisciplinary panel discuss the biological and clinical burden of obesity, emerging innovations in biotech and clinical practice, and patient experiences and societal factors.

Watch as expert panelists share insights into the most critical considerations for navigating early-phase clinical development and designing studies that reliably translate mechanisms into meaningful patient outcomes.

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