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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![Leukodystrophy Awareness Month-GettyImages-2166836167]( "The Preclinical Gauntlet: Securing Capital And Designing Regulatory Pathways For Ultra-Rare ASO Drugs (Pt. 2)")
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.
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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.
CLINICAL TRIALS VIDEOS
Capstan Therapeutics' CEO, Laura Shawver, Ph.D., talks us through the company's in vivo CAR therapies and what led to the in vivo cell engineering breakthrough that has been decades in the making. Shawver explains the promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo. She also details best practices to garner funding and investment partnership.
Hosted by Cell & Gene Collaborative Director Anna Rose Welch, ARW on RNA puts a creative spin on the emerging mRNA + RNA therapeutics industry. Here, in Episode 5, Welch dives into the separate — but strikingly similar — worlds of fly fishing and mRNA R&D. In particular, she explains how a fly fisherman’s strategies to catch a fish relate to the work of mRNA scientists as they strive to identify the most appropriate immunogenicity profiles for their mRNA vaccines or therapeutics.
Explore the evolving landscape of early-phase oncology clinical trials, including strategic insights to empower operations teams in optimizing study design, site selection, and recruitment.
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.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![Placebo_Article_AI]( "Navigating The Shift To Modular Digital Asset Architectures")
Decoupling metadata from core content creates agile workflows, enabling seamless asset reuse across global channels while maintaining technical precision.
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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.
