CLINICAL TRIALS ARTICLES
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![GettyImages-1358847958]( "From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion")
From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA ExpansionObviously, 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.
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![DNA helix model, clinical medicine-GettyImages-2280122904]( "Beyond Protein Targets: Why The Regulatory Genome Could Redefine RNA Medicine (Pt. 1)")
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.
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![GettyImages-480155972_Leukodystrophy]( "Building An ASO For TUBB4A-Related Leukodystrophy (Pt. 1)")
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.
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![AI, artificial intelligence, dna research-GettyImages-2192601384]( "Bridging The Atlantic: A European Executive's Guide To Entering The U.S. RNA Market (Pt. 1)")
Bridging The Atlantic: A European Executive's Guide To Entering The U.S. RNA Market (Pt. 1)A practical roadmap for European biotech leaders navigating FDA expectations, market entry, and the strategic decisions that shape U.S. expansion.
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![DNA Detection-GettyImages-618341764]( "The Missing Partners In microRNA Silencing: What QKI Reveals About RNA Regulatory Complexity")
The Missing Partners In microRNA Silencing: What QKI Reveals About RNA Regulatory ComplexityNew research suggests microRNA silencing depends on RNA-binding protein partners like QKI, revealing a more complex regulatory network shaping gene expression.
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![Emergency medicine-GettyImages-2055655561]( "Targeting The Uncharted Heart: Why The Next Era Of siRNA Belongs In Cardiology")
Targeting The Uncharted Heart: Why The Next Era Of siRNA Belongs In CardiologyDuring 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.
CLINICAL TRIALS VIDEOS
BioNTech’s Ben Muir shares a high-level overview of the LNP preclinical research advances that are the most exciting/promising today — while also reminding us of a few important caveats that accompany early-stage research.
Watch as expert panelists share insights into the most critical considerations for navigating early-phase clinical development and designing studies that reliably translate mechanisms into meaningful patient outcomes.
Watch this presentation to equip stakeholders with actionable strategies and a comprehensive understanding of the evolving clinical trial management landscape in the context of ICH guidelines.
In this video, Russell Johnson, VP Formulation Research for RVAC Medicines, and Aalok Shah, Director of Formulation & Drug Delivery for Strand Therapeutics, outline several examples of foundational research that could provide insights into how we attain more specific cellular uptake of LNPs.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![cell-gene-therapy-GettyImages-1407359737]( "Unmatched Beginnings For Late-Phase Success")
Early research choices shape outcomes. Focusing on reproducibility, scalability, regulatory fit, and supply stability early cuts risk, prevents delays, and supports smoother development progress.
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![GettyImages-2190243697-modern-lab-researching]( "DPCR, qPCR, And RT-qPCR: Advantages And Challenges In Development")
Discover how the right PCR strategy, combined with thoughtful assay design and validation, can strengthen confidence in your molecular research and clinical testing workflows.
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![GettyImages-1054148730-clinical-outsourcing-partner-FSP]( "Price Matters - Why Dash Believes In Transparency")
Opaque pricing slows decisions and erodes trust. Transparency clarifies cost drivers, improves budgeting, enables confident sourcing, and strengthens long-term R&D partnerships.
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![Single-Vendor CDMOs Bring Speed And Cost Savings To The Table]( "The Bioanalysis CRO Selection Checklist Every Sponsor Should Use Before Signing An MSA")
Choosing a CRO requires more than proposals. Assess reliability, automation, GLP rigor, scientific engagement, pricing clarity, and capacity to find partners who deliver defensible data on real timelines.
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![GettyImages-1333253892-laboratory-scientist-regulatory-compliance]( "GLP Compliance In A Modern Lab: What Is Actually Required?")
GLP compliance isn’t binary. Real defensibility depends on rigorous QA, validated electronic systems, and operational maturity — not just a label.
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![CGT-cro-GettyImages-2256752073]( "What Sponsors Get Wrong When Selecting A CRO")
Gene therapy immunogenicity requires platforms, judgment, and regulatory fluency beyond ADA norms. Relying on biologics criteria creates gaps; CGT expertise ensures reliable, interpretable results.
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![cell-therapy-GettyImages-1494029118]( "Why CGT Programs Need A Different CRO Mindset")
Advanced therapies need bioanalysis partners built for complexity, not scale. CGT expertise, specialized platforms, and flexible timelines ensure dosing and safety data traditional CROs can’t support.
