CLINICAL TRIALS ARTICLES
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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.
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![Investment in medical and pharma business GettyImages-1299804755]( "2026 RNA Investment Landscape: Recent Deals, IPOs, And Venture Trends")
2026 RNA Investment Landscape: Recent Deals, IPOs, And Venture TrendsAs RNA investing matures, capital is flowing toward delivery, durability, and regulatory credibility. This column breaks down the deals, IPO signals, and venture theses shaping RNA’s 2026 winners—and why.
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![Vector Retro Astronaut Team Exploring a Planet on a Jetpack-GettyImages-2186580479]( "A New Space Race: Reclaiming Efficiency In The mRNA Industry")
A New Space Race: Reclaiming Efficiency In The mRNA IndustryIt goes without saying that efficiency in drug development is tantamount — especially when it comes to getting much-needed clinical data. So, when I asked Allan Shaw about the ongoing trends he anticipates seeing more of in 2026, he immediately pointed to the increasing prominence of overseas development, particularly in nations like Australia and China.
CLINICAL TRIALS VIDEOS
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.
Hosted by Cell & Gene Collaborative's Director Anna Rose Welch, ARW on RNA puts a creative spin on the emerging mRNA + RNA therapeutics industry. Here, in Episode 1, Welch draws a few connections between the magical world of Harry Potter and the challenging scientific work mRNA companies are doing today to explore and/or broaden mRNA's therapeutic potential in a variety of indications.
The Advancing RNA Live panelists share several inconsistencies they’ve encountered working with global regulators, as well as on which aspects they’re seeing regulatory agencies applying greater scrutiny in mRNA development today.
Among the more prevalent genetic conditions, Duchenne muscular dystrophy affects an estimated one in 3,500 male births worldwide. It's caused by mutations of the DMD gene, which regulates the production of a protein called dystrophin. Approved DMD treatments haven't demonstrated strong clinical outcomes, but James McArthur, Ph.D. and his team at PepGen are seeking to change that with a pipeline of disease-modifying peptide-conjugated oligonucleotide candidates derived from the company's Enhanced Delivery Oligonucleotide platform. The Business of Biotech caught up with Dr. McArthur at PepGen's Cambridge headquarters to learn more.
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 Compliant Actually Requires In 2025")
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.
