CLINICAL TRIALS ARTICLES
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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.
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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.
CLINICAL TRIALS VIDEOS
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.
In this episode of the Cell & Gene podcast, Host Erin Harris speaks with Alicia Zhou, Ph.D., CEO of the Cancer Research Institute (CRI), about CRI’s mission to advance cancer immunotherapy and the critical role of federal funding, particularly from the NIH, in supporting basic and translational cancer research.
Gain insight into why certain regions offer strategic advantages, from regulatory flexibility to faster enrollment, and how to effectively integrate them into your development strategy.
PepGen, a Cambridge, MA-based biotech, is advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. SVP Head of Clinical Development Dr. Michelle Mellion explains PepGen's trial of PGN-EDO51, the company’s lead product candidate for the treatment of DMD patients whose mutations are amenable to an exon 51 skipping approach. She also explains how the delivery of oligonucleotide therapies differ from other techniques in genetic medicine, and much 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.
