CLINICAL TRIALS ARTICLES

• Inside the In Vivo CAR-T Boom: Where Does mRNA Fit In?

  I had the chance to sit down with ParcelBio’s CEO David Weinberg to learn more about the emerging market dynamics in the in vivo CAR-T space, with a particular focus on the challenges facing RNA biotechs looking to navigate this up-and-coming therapeutic class.

• What Baby KJ Taught Us About Patient & Physician Communication

  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. 

• 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.