RNA Clinical Trials Articles

31. Excision BioTherapeutics & Guide RNA 7/27/2023

    Excision’s CEO Daniel Dornbusch to dig into its CRISPR-based therapy, EBT-101, as well as Guide RNA (gRNA) and why it is critical to EBT-101’s success. 

32. Targeting RNA With Small Molecules 7/25/2023

    Beyond mRNA's dogmatic role as the intermediate in gene expression, non-coding RNA play essential roles in transcription regulation, maturation, and translation. This article explores the potential of RNA as a clinically validated avenue for drug development, methods for design and discovery, and implications for the future of medicine.

33. Targeting Von Willebrand Factor With An RNA Aptamer To Treat Stroke 6/6/2023

    Developing new treatments for acute ischemic stroke that can quickly and safely intervene at the right place and time has been a challenging endeavor. Today, the situation is changing due to technological developments related to stroke and recent advances in RNA-based medicines.

34. Where Fly-Fishing Meets mRNA: The Art Of mRNA Immunogenicity 5/22/2023

    In the final installment of this two-part series comparing fly-fishing and mRNA immunogenicity, I summarize some of the basic immunology-centric underpinnings of our mRNA development efforts and the enduring immunogenicity questions and concerns shaping and re-shaping these efforts today.

35. What Fly-Fishing Can Teach Us About mRNA Immunogenicity 5/17/2023

    In part one of this two-part article, I spell out some of the decisions fly-fishermen must make to “trick” a fish, and how these decisions are akin to those of the mRNA industry as we strive to successfully navigate “the enigmatic beast” that is the human immune system. 

36. Metaphorical mRNA: An Argument For (More) Creativity In RNA Therapeutics 1/25/2023

    In putting together my mRNA outlook presentation for November's 2022 AGC CDMO Summit, I realized there is one opportunity that should be top of mind for the RNA therapeutics industry in the years to come. We all, regardless of our functional area in the industry (i.e., manufacturing, quality, R&D, commercial), need to figure out how we can make this important science more accessible to populations around the world. 

37. Defining The RNA Therapeutics Industry In 2023 1/16/2023

    Four executives outline which industry challenges, strategic considerations, and opportunities should be top of mind as RNA therapeutics companies establish their to-do lists for 2023.

38. mRNA & RNA Therapeutics In 2023: Where Do We Go From Here? 12/7/2022

    To figure out what the future holds for companies in the RNA sector of the ATMP space, I sat down with four executives to discuss their wildest hopes, dreams, and expectations. As you’ll note, there is no shortage of work facing the RNA therapeutics industry in the year(s) ahead. But there’s also a lot for which we can hope.

39. DTx Pharma And The Near-Term Future Of RNA Therapeutics 11/21/2022

    To learn more about the near-term future of RNA therapeutics, the advantages of short interfering RNA (siRNA) over other RNA therapeutics and more, I caught up with DTx Pharma's CEO Arthur Suckow.

40. The Promising Future Of Oligonucleotides 10/31/2022

    Episode 35 of Cell & Gene: The Podcast features Dr. Michelle Mellion, PepGen’s SVP Head of Clinical Development at Cambridge, MA-based biotech PepGen. In it, Dr. Mellion talks all about the company’s oligonucleotide therapeutics that were developed to treat severe neuromuscular and neurologic diseases, such as Duchenne Muscular Dystrophy (DMD).