CLINICAL TRIALS ARTICLES
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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.
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![Business support, teamwork-GettyImages-2267605245]( "The RNA Ties That Bind: 3 Takeaways From The OPT Congress")
The RNA Ties That Bind: 3 Takeaways From The OPT CongressHere, 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.
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![Global health safety regulation-GettyImages-2245658628]( "How Informal FDA Feedback Is Quietly Driving RNA Capital Allocation")
How Informal FDA Feedback Is Quietly Driving RNA Capital AllocationSubtle 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.
CLINICAL TRIALS VIDEOS
Explore the behind-the-scenes best practices of the company implemented that led to the approval of the world’s first saRNA vaccine in Japan.
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.
Nutcracker Therapeutics is a preclinical biopharma company developing multimodal RNA-based therapeutics for HPV-driven tumors, T-cell lymphoma, and Genitourinary tumors. On this episode of the Business of Biotech, Chief Business Officer Geoff Nosrati, Ph.D. offers a comprehensive view of the RNA-based therapeutics landscape and sheds light on what he perceives as an advantageous position at Nutcracker — the capacity to manufacture RNA in-house. If you're curious about the expanding science behind RNA-based therapeutics and their application in biotech, this conversation with a scientist-turned-chief business officer (Nosrati earned his Ph.D. in Biochemistry and Molecular Biology) is a can't-miss episode.
Dr. Francois Vigneault, Co-Founder, President, and CEO at Shape Therapeutics joins Cell & Gene: The Podcast's Erin Harris to discuss how RNA technologies are shaping the future of gene therapy. They also discuss AI's role in gene therapy as well as how the CGT sector is benefiting from RNA, AI, and synthetic biology.
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.
