RNA Clinical Trials Videos

31. 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.

32. 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.

33. Overcoming Regulatory, Safety/Efficacy, And Toxicity Hurdles 11/28/2022

    In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, discuss what industry challenges should be top of mind for experts as it relates to the regulatory paradigm, toxicity/safety, and in vivo modeling.

34. 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.

35. 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.

36. What Innovations Could Help Us Overcome Preclinical Model Limitations For RNA-LNPs? 12/4/2024

    In this Advancing RNA Live clip, BioNTech’s Ben Muir and Hopewell Therapeutics’ Kate Zhang share their perspectives on the most exciting novel screening methods, innovative preclinical models, and next-gen technologies they hope can/will eventually help us bridge the translation gap between preclinical models and the clinic.

37. Adapting Clinical Strategies For Rapid Response 12/5/2024

    This presentation aims to equip sponsors, researchers, and stakeholders with the tools to enhance trial resilience and ensure continuity in today’s unpredictable global landscape.

38. PepGen's SVP Head Of Clinical Development On Delivery Of Oligonucleotide Therapies 10/27/2022

    PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much more.

39. 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.