RNA Clinical Trials Videos

1. PepGen's SVP Head Of Clinical Development On Delivery Of Oligonucleotide Therapies 10/27/2022

   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.

2. R&D & CMC Priorities For LNPs Beyond Cell-Specific Delivery 12/4/2024

   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.

3. Early Phase Excellence: Navigating B Cell Therapies In Autoimmune Conditions 12/2/2025

   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.

4. Proactive Partnerships: Engaging FDA And EMA During mRNA Process Development 6/24/2025

   Panelists Tiffany Lucas and Ulrike Jägle address an audience question about when and how to approach the regulatory agencies for mRNA process development. In addition to listing the different FDA and EMA meeting opportunities available to sponsors, Jägle reminds us of one critical best practices sponsors shouldn’t forget when approaching the agencies.

5. Precision ADVANCE: Unravelling The Complexity Of CGTx Development 10/12/2021

   Need help unravelling the complexity of CGTx development? Four experts explain Precision ADVANCE’s unique approach. Spend 50 seconds with Anshul Mangal, Deb Phippard, Megan Liles, and Philip Cyr.

6. Achieving mRNA-LNP Delivery Beyond The Liver 11/28/2022

   In this segment, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, discuss which ongoing industry efforts to improve LNPs and the RNA/mRNA cargo they're watching closely that could help the RNA industry move beyond hepatic delivery.

7. What Innovations Could Help Us Overcome Preclinical Model Limitations For RNA-LNPs? 12/4/2024

   In this Advancing RNA Live clip, BioNTech’s Ben Muir and Hopewell Therapeutics’ Kate Zhang share their perspectives on the most exciting novel screening methods, innovative preclinical models, and next-gen technologies they hope can/will eventually help us bridge the translation gap between preclinical models and the clinic.

8. Oligonucleotide Opportunities in DMD with PepGen's James McArthur, Ph.D. 7/25/2022

   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.

9. Overcoming Regulatory, Safety/Efficacy, And Toxicity Hurdles 11/28/2022

   In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, discuss what industry challenges should be top of mind for experts as it relates to the regulatory paradigm, toxicity/safety, and in vivo modeling.

10. Accelerating Clinical Development In China & The US 9/22/2022

    Discover how the clinical research bridge between China and the US supports accelerated clinical development from early to late phase trials while ensuring the inclusion of MRCT data packages in future regulatory applications.