RNA Clinical Trials Videos

1. 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.

2. Translation Of RNA Nanomedicines From Design To Clinical 4/14/2022

   mRNA therapeutics are applicable to cancer immunotherapies, infectious diseases, and other indications that require protein replacement therapy or antibodies.

3. 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.

4. The Intersection Of RNA, AI, And Synthetic Biology With Shape Therapeutics' Dr. Francois Vigneault 7/18/2023

   Dr. Francois Vigneault, Co-Founder, President, and CEO at Shape Therapeutics joins Cell & Gene: The Podcast's Erin Harris to discuss how RNA technologies are shaping the future of gene therapy. They also discuss AI's role in gene therapy as well as how the CGT sector is benefiting from RNA, AI, and synthetic biology.

5. Who's In Charge? How To Ensure Effective Trial Oversight, Leadership 3/19/2025

   Gain valuable insights into the role of sponsor-side Clinical Operations leadership, the risks of over-relying on CROs, and key strategies for fostering collaboration between sponsors and partners.

6. The Scientific Underpinnings Of mRNA/LNPs 11/28/2022

   In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery, Strand Therapeutics, share their thoughts on the scientific-research and CMC-related reasons that have made the lipid nanoparticle the “darling” of the delivery world for mRNA/RNA products today.

7. 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.

8. 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.

9. 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.

10. 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.