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
This presentation aims to equip sponsors, researchers, and stakeholders with the tools to enhance trial resilience and ensure continuity in today’s unpredictable global landscape.
Tessera Therapeutics' CEO, Dr. Michael Severino, joins Host Erin Harris to discuss Gene Writing, the biotech's genome engineering technology that writes therapeutic messages into the genome to treat diseases at their source. Severino discusses why delivery has always been a challenge for gene editing technologies and why Gene Writing is different when it comes to efficient delivery.
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.
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.
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.
