RNA Clinical Trials Videos

1. 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.

2. 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.

3. 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.

4. A New Approach to Genome Engineering with Tessera Therapeutics' Dr. Michael Severino 9/11/2024

   Tessera Therapeutics' CEO, Dr. Michael Severino, joins Host Erin Harris to discuss Gene Writing, the biotech's genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source. Severino discusses why delivery has always been a challenge for gene editing technologies and why Gene Writing is different when it comes to efficient delivery.

5. 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.

6. 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.

7. 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.

8. 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.

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. 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.