CLINICAL TRIALS ARTICLES

CLINICAL TRIALS VIDEOS

Watch this presentation to equip stakeholders with actionable strategies and a comprehensive understanding of the evolving clinical trial management landscape in the context of ICH guidelines.

Gain an understanding of how an advanced nuclease portfolio is designed to support CRISPR-based cell and gene therapy development, providing scientists with the best solutions for therapeutic success.

PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much more.

It goes without saying that achieving cell-specific delivery is the most often discussed challenge facing the RNA-LNP field near- and long-term. However, as these three LNP experts share in this Advancing RNA Live clip, they have a whole list of additional R&D and CMC priorities we must address/accomplish to improve RNA-LNPs’ efficacy in personalized and gene therapy approaches.

ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS