CLINICAL TRIALS ARTICLES
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![T-Cells Work to Fight Cancer, Immunotherapy, CAR T-cell therapy-GettyImages-1317697233]( "Overcoming CMC Challenges In The Development Of Conjugated LNP Platforms For In Vivo CAR-T Therapy")
Overcoming CMC Challenges In The Development Of Conjugated LNP Platforms For In Vivo CAR-T TherapyOvercoming CMC hurdles is key to advancing conjugated LNP platforms for in vivo CAR-T therapy, enabling scalable, precise mRNA delivery to T cells while maintaining stability, potency, and regulatory compliance.
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![Bubble burst-GettyImages-1215335273]( "Beyond the Hype: Industry Experts on the Reality of mRNA in Cancer Care")
Beyond the Hype: Industry Experts on the Reality of mRNA in Cancer CareWhen I asked a panel of experts at RNA Leaders why mRNA was best positioned to shine in oncology, I was met with a few expected talking points and — better yet — a few I wasn’t expecting.
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![Magnifying Glass, explain mechanism of mRNA vaccine-GettyImages-1903242094]( "Tackling Challenges In RNA Stability And Organ Targeting")
Tackling Challenges In RNA Stability And Organ TargetingRNA therapeutics are reshaping medicine, but instability and limited organ targeting still pose challenges. Discover how next-gen lipid nanoparticles and smart delivery systems are advancing RNA precision.
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![Telemedicine, human Brain recovery-GettyImages-1397374666]( "Developing Exosome-Based RNA Therapeutics For CNS Regeneration")
Developing Exosome-Based RNA Therapeutics For CNS RegenerationInside the effort to use exosomes as natural nanocarriers that deliver RNA therapeutics safely across the blood–brain barrier to heal damaged neural tissue.
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![Wealth-GettyImages-869781702]( "Bridging RNA Innovation: A CEO's Guide To European Expansion")
Bridging RNA Innovation: A CEO's Guide To European ExpansionFor RNA biotechs, Europe isn't just the next market — it's the ultimate test of strategy. Here's what CEOs need to know.
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![Utility teamwork-GettyImages-2211320040]( "From Misinformation To Medicine: Forging Bipartisan Support to Reverse Anti-mRNA Policy")
From Misinformation To Medicine: Forging Bipartisan Support to Reverse Anti-mRNA PolicyIn this article, I’ll share some of the progress AMM has made and the barriers the organization/our industry is still facing in our efforts to reverse the policy decisions that have been made against mRNA today. Throughout the panel, the speakers also shared their thoughts on the types of messaging we should be considering and/or have started to see making an impact.
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.
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.
Gain an understanding of how an advanced nuclease portfolio is designed to support CRISPR-based cell and gene therapy development, providing scientists with the best solutions for therapeutic success.
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.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![GettyImages-2006338804 conference]( "ESMO Congress 2025: Highlights From Berlin")
Turn the insights from this year’s ESMO into action by partnering to accelerate innovation through smarter trial design, stronger biomarker planning, and truly global collaboration.
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![GettyImages-2152658176 oncology, doctor, patient]( "How Are Therapeutic Radiopharmaceuticals Advancing Precision Oncology?")
By directing radioactive isotopes straight to diseased tissue, these agents deliver potent, localized treatment while sparing healthy cells — a major leap forward for patients with metastatic or treatment-resistant cancers.
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![Doctors board room meeting-GettyImages-1365555907]( "Highlights From The 2025 International Hepatitis B Meeting")
For biotech and pharmaceutical teams, the core message was clear: progress is accelerating, but success still depends on smart study design, fit-for-purpose endpoints, and operational execution.
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![GettyImages-2010746888-clinical-meeting-tablet-collaboration]( "Therapeutic Radiopharmaceuticals: Clinical Research, CRO Enablers (2025)")
Accelerate the future of precision oncology by driving innovation, optimizing clinical development, and expanding access to targeted therapies that transform patient outcomes.
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![AI, artificial intelligence-GettyImages-2205274485]( "Using AI Tools In Hepatology Drug Development")
Explore how AI can help overcome key challenges in hepatology trials — from site selection and patient matching to data integration and engagement — while ensuring diversity, privacy, and transparency.
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![dna, rna molecules-GettyImages-537327406]( "The Evolving RNA Landscape: Navigating Science, Strategy, And Global Markets")
Learn how to streamline development, reduce costs, and accelerate the path to market for next-generation RNA therapies.
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![GettyImages-871431000 doctor partner]( "Five Questions Biotechs Should Ask Before Selecting A Bioanalytical CRO")
In a landscape where speed, quality, and trust are inseparable, choosing a CRO that shares your ambition is not optional; it is essential to survival and success.
