RNA Clinical Trials Videos

1. 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.

2. 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.

3. 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.

4. Michelangelo Meets mRNA | ARW on RNA Ep04 5/4/2023

   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 4, Welch explores the parallels that exist between the oft-overlooked history of Michelangelo's David and the mRNA therapeutics sector's own R&D history, as well as where the mRNA space hopes to move in the future.

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. 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.

7. 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.

8. 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.

9. 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.

10. 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.