RNA Clinical Trials Videos

  1. Goldilocks & The 3…CQAs? Finding "Just Right" With LNP Optimization 12/4/2024

    Three LNP experts from Sanofi, Hopewell Therapeutics, and BioNTech collectively discuss how far we’ve come in understanding the structure of our LNPs while also shedding light on the variables that have the greatest potential of causing shifts in our products’ structure/function.

  2. Harry Potter Meets mRNA | ARW On RNA Ep01 2/1/2023

    Hosted by Cell & Gene Collaborative's Director Anna Rose Welch, ARW on RNA puts a creative spin on the emerging mRNA + RNA therapeutics industry. Here, in Episode 1, Welch draws a few connections between the magical world of Harry Potter and the challenging scientific work mRNA companies are doing today to explore and/or broaden mRNA's therapeutic potential in a variety of indications.

  3. 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. 

  4. 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.

  5. RNA-Based Therapeutics With Nutcracker Therapeutics' Dr. Geoff Nosrati 11/21/2022

    Nutcracker Therapeutics is a preclinical biopharma company developing multimodal RNA-based therapeutics for HPV-driven tumors, T-cell lymphoma, and Genitourinary tumors. On this episode of the Business of Biotech, Chief Business Officer Geoff Nosrati, Ph.D. offers a comprehensive view of the RNA-based therapeutics landscape and sheds light on what he perceives as an advantageous position at Nutcracker — the capacity to manufacture RNA in-house. If you're curious about the expanding science behind RNA-based therapeutics and their application in biotech, this conversation with a scientist-turned-chief business officer (Nosrati earned his Ph.D. in Biochemistry and Molecular Biology) is a can't-miss episode.

  6. Development And Regulatory Strategy For US And China 9/22/2022

    This webinar discusses the expansion of International Market for Ensartini and RNAi Therapeutics in Oncology, from Skin Cancers to Liver Cancers.

  7. 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.

  8. 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.

  9. 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.

  10. 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.