RNA Clinical Trials Videos

1. 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.

2. A Snapshot Of The RNA Industry & Its Regulatory Progress 5/10/2024

   The experts on this Advancing RNA Live panel share their takeaways from several audience poll questions revealing which RNA molecules are getting the lion’s share of attention/development today.

3. 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.

4. Accelerating Development In Novel & Advanced Oncology Therapies 6/15/2023

   Hear from a panel of industry experts on dissecting the shift in early-phase oncology design, focusing on Bayesian Data.

5. From Crisis To Confidence: Ensuring Smooth Regulatory Submissions 10/8/2024

   Explore some of the major challenges that medical writing submission specialists face today and strategies that can consistently set the stage for success.

6. Q&A On LNP Cell-Specific Delivery 11/28/2022

   In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery for Strand Therapeutics, outline several examples of foundational research that could provide insights into how we attain more specific cellular uptake of LNPs.

7. How A Sponsored CMC Platform Accelerates mRNA Projects Into The Clinic 1/31/2023

   Developing a client-sponsored Target Product Profile (TPP) and Analytical Target Profile (ATP) is critical to move your drug product to clinic efficiently and meet acceptance criteria.

8. 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.

9. 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.

10. 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.