CLINICAL TRIALS ARTICLES
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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.
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![Ribosome, biological cell constructing mRNA molecule-GettyImages-1352440366]( "Beyond Hairpins: A New Translational Repression Assay Expands RNA-Binding Protein Research")
Beyond Hairpins: A New Translational Repression Assay Expands RNA-Binding Protein ResearchResearchers expanded translational repression assays beyond RNA hairpins, enabling scalable study of linear RNA-protein interactions in living cells.
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![genetic engineering and gene manipulation, CRISPR technology, genome editing, molecular biotechnology, genetic science research, gene therapy-GettyImages-2196575232]( "From Supporting Component to Strategic Driver: The Evolving Role of gRNA in CRISPR Therapeutics")
From Supporting Component to Strategic Driver: The Evolving Role of gRNA in CRISPR TherapeuticsGuide 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.
CLINICAL TRIALS VIDEOS
It goes without saying that achieving cell-specific delivery is the most often discussed challenge facing the RNA-LNP field near- and long-term. However, as these three LNP experts share in this Advancing RNA Live clip, they have a whole list of additional R&D and CMC priorities we must address/accomplish to improve RNA-LNPs’ efficacy in personalized and gene therapy approaches.
mRNA therapeutics are applicable to cancer immunotherapies, infectious diseases, and other indications that require protein replacement therapy or antibodies.
Developing a client-sponsored Target Product Profile (TPP) and Analytical Target Profile (ATP) is critical to move your drug product to clinic efficiently and meet acceptance criteria.
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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![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.
