FORMULATION ARTICLES
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![neuroscience, neuromuscular disorders, pain signals, rehabilitation-GettyImages-2280116713]( "Why Delivery Is Defining The Next Era Of Neuromuscular Therapeutics")
Why Delivery Is Defining The Next Era Of Neuromuscular TherapeuticsEmerging therapies are being designed to safely deliver oligonucleotides to disease-relevant tissues. Delivery is no longer a technical hurdle to overcome but a strategic lever.
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![intelligent technology interface, robot chat-GettyImages-2279572840]( "Analytical Platforms: The Foundation Of The Next Generation Of RNA Therapeutics")
Analytical Platforms: The Foundation Of The Next Generation Of RNA TherapeuticsRNA therapeutics are entering a new era where analytical platforms drive scalability, regulatory confidence, manufacturing flexibility, and the development of increasingly complex products.
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![nucleic acid, DNA, RNA, purified protein-GettyImages-1350828873]( "A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA Products")
A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA ProductsNIST research introduces a gentle SEC method that separates dsRNA impurities from IVT mRNA by tweaking buffer pH, achieving 81.8% target recovery without transcript damage.
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![GettyImages-1316503044-dna-strand-double-helix-gene-editing]( "The Second Holy Grail Of Gene Editing: Large Knock-Ins")
The Second Holy Grail Of Gene Editing: Large Knock-InsExploring the race to achieve large gene knock-ins, emerging editing platforms, and how overcoming this challenge could unlock therapies for many inherited diseases.
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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.
FORMULATION VIDEOS
mRNA therapeutics are applicable to cancer immunotherapies, infectious diseases, and other indications that require protein replacement therapy or antibodies.
A connected development pathway supports smoother scale-up and greater consistency for LNP-based therapeutics. Explore how integrated expertise across key stages helps teams accelerate progress.
Together, Life Edit Therapeutics’ April Sena and Sanofi’s Troiano share their perspectives on raw material quality & IP-related considerations for managing the supply chain for next-generation RNA modalities.
It goes without saying that achieving cell-specific delivery is the most often discussed challenge facing the RNA-LNP field near- and long-term. However, as these three LNP experts share in this Advancing RNA Live clip, they have a whole list of additional R&D and CMC priorities we must address/accomplish to improve RNA-LNPs’ efficacy in personalized and gene therapy approaches.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![GettyImages-1437856375-lnp-lipid-nanoparticle]( "Regulatory Considerations For Excipients Used In Lipid Nanoparticles")
Review the regulatory classification of LNPs, liposomes, and novel excipients as well as specific requirements outlined in regulatory guidance documents and strategies to navigate emerging challenges.
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![GettyImages-597935610 cell and gene]( "Delivering The Promise Of Gene Therapy")
Gene therapies rely on efficient, precise methods of delivering genetic material. Examine how combining polymeric nanoparticles with CRISPR therapies is increasing the potential for improved treatment.
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![GettyImages-1409578453-mRNA-LNP-lipid-nanoparticle-RNA therapy]( "Optimized And Scalable LNP Composition For RNA Vaccine Delivery")
Explore results from a study screening novel LNP formulations for physicochemical attributes, in vitro potency, and in vivo immunogenicity in both SARS-CoV-2 and influenza vaccine applications.
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![Treatment with Nanoparticles Genetic therapy GettyImages-805434264]( "Process Development Considerations For RNA-LNP Therapeutics")
Here, we highlight three important process considerations across the RNA-LNP manufacturing workflow which includes limit size behavior, in-line dilution and downstream tangential flow filtration (TFF).
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![GettyImages-1087219020 Scientists wearing protective coveralls working in manufacturing laboratory]( "Insights Into GMP Manufacturing Of RNA-LNP Drug Products")
Explore key considerations for manufacturing RNA-LNP drug products surrounding process and analytical development, automation, environmental monitoring, and strategic collaborations.
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![Outsourcing RNA Feature Image]( "Outsourcing RNA-LNP Production: A Key To Competitive Success")
Manufacturing RNA-containing LNPs demands specialized expertise. Explore some of the challenges of RNA-LNP drug manufacturing and the advantages of outsourcing RNA-LNP formulations.
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![Cytiva-602047-2. Translation of RNA]( "Translation Of RNA Medicines From Design To Clinic")
Understand the advantages and challenges of lipid-based delivery systems for successful translation of RNA into clinical development.
