CLINICAL TRIALS ARTICLES

• What Baby KJ Taught Us About mRNA COGS

  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.”

• What Baby KJ Taught Us About Sustainable mRNA Development

  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. 

• From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion

  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.

• The Preclinical Gauntlet: Securing Capital And Designing Regulatory Pathways For Ultra-Rare ASO Drugs (Pt. 2)

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

• Beyond Protein Targets: Why The Regulatory Genome Could Redefine RNA Medicine (Pt. 1)

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

• Building An ASO For TUBB4A-Related Leukodystrophy (Pt. 1)

  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.