RNA Clinical Trials Articles

1. 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.

2. 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.

3. 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.

4. 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.

5. Beyond Hairpins: A New Translational Repression Assay Expands RNA-Binding Protein Research 5/26/2026

   Researchers expanded translational repression assays beyond RNA hairpins, enabling scalable study of linear RNA-protein interactions in living cells.

6. From Supporting Component to Strategic Driver: The Evolving Role of gRNA in CRISPR Therapeutics 4/21/2026

   Guide RNA is evolving from a simple targeting reagent into a complex, engineered molecule shaped by expanding CRISPR modalities, increasing length and modification demands, and tighter integration with manufacturability, analytical control, and regulatory expectations.

7. The RNA Ties That Bind: 3 Takeaways From The OPT Congress 4/1/2026

   Here, I unpack three takeaways I had from the OPT Congress, each of which I’d argue are indicative of the interconnectedness across the genetic medicines field.

8. How Informal FDA Feedback Is Quietly Driving RNA Capital Allocation 3/19/2026

   Subtle FDA signals — especially in pre-IND talks — are quietly shaping RNA investment. Early regulatory feedback on CMC and comparability now drives how investors assess risk and allocate capital.

9. 2026 RNA Investment Landscape: Recent Deals, IPOs, And Venture Trends 2/22/2026

   As RNA investing matures, capital is flowing toward delivery, durability, and regulatory credibility. This column breaks down the deals, IPO signals, and venture theses shaping RNA’s 2026 winners—and why.

10. A New Space Race: Reclaiming Efficiency In The mRNA Industry 1/27/2026

    It goes without saying that efficiency in drug development is tantamount — especially when it comes to getting much-needed clinical data. So, when I asked Allan Shaw about the ongoing trends he anticipates seeing more of in 2026, he immediately pointed to the increasing prominence of overseas development, particularly in nations like Australia and China.