RNA Clinical Trials Videos

31. Approving The World's First saRNA Vaccine: Arcturus' Regulatory Experience 5/10/2024

    Explore the behind-the-scenes best practices of the company implemented that led to the approval of the world’s first saRNA vaccine in Japan.

32. 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.

33. 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.

34. 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.

35. 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.

36. 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.

37. 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.

38. 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.    

39. 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.

40. 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.