RNA Clinical Trials Articles

  1. What Baby KJ Taught Us About Patient & Physician Communication 9/1/2026

    Over the past few weeks, I’ve been sharing my takeaways from the overall event, as well as from a specific panel featuring JHU’s Jeff Coller, CHOP’s Rebecca Ahrens-Nicklas, and Plowshare Therapies’ Kevin Strauss. Together, these three discussed the biggest learnings and takeaways from the miraculous Baby KJ experience that we very much hope to replicate in the future. As you can likely imagine, patient and physician communication played a large role in the discussion at hand. 

  2. What Baby KJ Taught Us About mRNA COGS 8/25/2026

    Here, in part 2 of this three-part series, I share a few details that came to light about the COGS behind Baby KJ’s therapy production. Obviously, we know why it’s important to keep these figures in mind as we move forward; but I particularly liked Strauss’ reminder that it’s our job to “think systematically about how we’re going to create a safety net of sustainable, accessible product that can be widely distributed at an affordable cost.”

  3. What Baby KJ Taught Us About Sustainable mRNA Development 8/18/2026

    Of course, these conversations are rarely simple, and the learnings are multifaceted. In the following three-part series, I’ll share my 3 biggest takeaways from the discussion between Coller, Ahrens-Nicklas, and Strauss. Here in part 1, I’ll start by parsing out the learnings about drug development we can take away from the overall heroic efforts to dose Baby KJ. 

  4. From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion 8/8/2026

    Obviously, the transition from RNA vaccines to therapeutics is an “expansion” all on its own; but to do this successfully, we need to expand our science, our products’ deliverability, and our patient access. In the following article, I highlight a few of the best practices and/or innovations that came up in conversation during this event that will play a significant role in helping us expand mRNA’s reach, both scientifically and therapeutically.

  5. The Preclinical Gauntlet: Securing Capital And Designing Regulatory Pathways For Ultra-Rare ASO Drugs (Pt. 2) 8/7/2026

    SynaptixBio CEO Dan Williams explains why funding, regulatory strategy, toxicology, and disciplined execution — not just science — determine success in advancing ultra-rare ASO therapies.

  6. Beyond Protein Targets: Why The Regulatory Genome Could Redefine RNA Medicine (Pt. 1) 8/4/2026

    Explore how the regulatory genome and lncRNAs are shifting RNA medicine beyond protein targets, enabling cell-state reprogramming to treat disease at its source.

  7. Building An ASO For TUBB4A-Related Leukodystrophy (Pt. 1) 7/28/2026

    SynaptixBio CEO Dan Williams explains how the company selected an ASO for TUBB4A leukodystrophy by balancing potency, safety, and translational rigor to advance a rare disease therapy.

  8. Bridging The Atlantic: A European Executive's Guide To Entering The U.S. RNA Market (Pt. 1) 7/13/2026

    A practical roadmap for European biotech leaders navigating FDA expectations, market entry, and the strategic decisions that shape U.S. expansion.

  9. The Missing Partners In microRNA Silencing: What QKI Reveals About RNA Regulatory Complexity 6/26/2026

    New research suggests microRNA silencing depends on RNA-binding protein partners like QKI, revealing a more complex regulatory network shaping gene expression.

  10. Targeting The Uncharted Heart: Why The Next Era Of siRNA Belongs In Cardiology 6/15/2026

    During our conversation, Gallagher walked me through what’s keeping her up at night as she leads the company and its first siRNA candidate toward an IND, elaborating on the role RNA can play in cardiomyopathies and identifying the unique challenges of developing RNA medicines today.