RNA Clinical Trials Videos

1. 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.

2. 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.

3. A Deeper Dive On Mitigating Financial Risk Before Selecting A Vendor 1/23/2024

   In part two of this webinar series, presenters focus on avoiding potential risks, such as unexpected budget changes and unplanned change orders, that could jeopardize the success of a clinical trial.

4. Fly Fishing Meets mRNA | ARW On RNA Ep05 7/12/2023

   Hosted by Cell & Gene Collaborative Director Anna Rose Welch, ARW on RNA puts a creative spin on the emerging mRNA + RNA therapeutics industry. Here, in Episode 5, Welch dives into the separate — but strikingly similar — worlds of fly fishing and mRNA R&D. In particular, she explains how a fly fisherman’s strategies to catch a fish relate to the work of mRNA scientists as they strive to identify the most appropriate immunogenicity profiles for their mRNA vaccines or therapeutics.    

5. ICH E6(R3): Practical Steps For Implementation 12/14/2023

   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.

6. 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.

7. 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.

8. The Scientific Underpinnings Of mRNA/LNPs 11/28/2022

   In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, share their thoughts on the scientific-research and CMC-related reasons that have made the lipid nanoparticle the “darling” of the delivery world for mRNA/RNA products today.

9. Who's In Charge? How To Ensure Effective Trial Oversight, Leadership 3/19/2025

   Gain valuable insights into the role of sponsor-side Clinical Operations leadership, the risks of over-relying on CROs, and key strategies for fostering collaboration between sponsors and partners.

10. Obesity, From The Bench To The Bedside 12/2/2025

    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.