RNA Clinical Trials Videos

31. 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.

32. Navigating The Early Oncology Landscape: Market Trends And Insights 5/14/2025

    Explore the evolving landscape of early-phase oncology clinical trials, including strategic insights to empower operations teams in optimizing study design, site selection, and recruitment.

33. 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.

34. 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.

35. Shaping Therapeutic Development With Cutting-Edge CRISPR Nucleases 6/4/2025

    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.

36. What Have We Learned About Our LNP's Function In Vitro In 2024? 12/4/2024

    BioNTech’s Ben Muir shares a high-level overview of the LNP preclinical research advances that are the most exciting/promising today — while also reminding us of a few important caveats that accompany early-stage research. 

37. Go/No Go Decisions Based On Early Phase Oncology Trials 9/22/2022

    In this webinar, gain insight into the latest regulatory and clinical developments in the field of oncology in China.

38. Capstan Therapeutics' CEO Laura Shawver On In Vivo Cell Engineering 11/10/2022

    Capstan Therapeutics' CEO, Laura Shawver, Ph.D., talks us through the company's in vivo CAR therapies and what led to the in vivo cell engineering breakthrough that has been decades in the making. Shawver explains the promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo. She also details best practices to garner funding and investment partnership.

39. 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.

40. 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.