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
Develop RNA-lipid nanoparticles (LNPs) in under 10 seconds for drug discovery and early preclinical development. The NanoAssemblr®️ Spark™ instrument enables rapid µL formulations to conserve limited and expensive ingredients with minimal waste.
Learn more about five key factors for advancing targeted in vivo LNP programs, from formulation and targeting strategies to bioanalytical readiness and scalability.
There are many different criteria for selecting a CDMO for your mRNA products. Arcturus’ Qian Ruan explains that a product’s stage of development and the specific needs inherent to that phase of development should be an important guide for companies today, especially given the expansion of the outsourcing sector and the wide range of capabilities.
Nutcracker Therapeutics’ Oreanna Thomas shares her takeaways from a pre-IND meeting for one of the company’s mRNA therapeutic candidates.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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![Lipid Nanoparticles GettyImages-1437855772]( "Rethink Cell Therapy With LNPs")
Explore how lipid nanoparticles are revolutionizing cell therapy workflows to enable the precise delivery of mRNA and gene editing tools for next-generation treatments like CAR T and HSC therapies.
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![Cryopreservation GettyImages-172640159]( "The Slow Drift Toward Variability And The Case For Standardization")
Small, early operational decisions accumulate over time, creating hidden variability that emerges at scale, making standardization essential for stability, reproducibility, and regulatory confidence.
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![GettyImages-1287846309-research-lab-computer-graph-data]( "Low-Volume Production For Screening High-Value Nanoparticle Materials")
A platform that harnesses microfluidic mixing has been demonstrated as a simple, robust, and scalable production method for LNPs encapsulating various types of nucleic acids.
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![GettyImages-1288118586 cell development]( "Expectations On The Pathway To GMP For Gene-Modified Cell Therapies")
Learn how a robust understanding of the RNP’s critical process parameters and CQAs is crucial for ensuring product consistency, enhancing patient safety, and maintaining regulatory compliance.
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![RNA abbreviation of ribonucleic acid-GettyImages-1453953422]( "Exploring Osmolality As A CQA Within RNA Therapeutics")
Discover how osmolality can be applied throughout the bioprocessing workflow for RNA, and recognize the importance of this sensitive measurement, from formulation optimization to cryopreservation techniques.
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![GettyImages-1341853306-logistics-network-globe-data-information]( "Defragmenting The Pre‑Clinical Supply Chain")
Fragmented pre‑clinical supply chains create avoidable delays and risks; unifying cryopreservation, logistics, and storage early improves consistency, reduces operational gaps, and supports smoother progression into clinical phases.
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![MRNA Technology-GettyImages-1366661187]( "Developing An mRNA-Encoded Antibody Platform To Accelerate Therapies To Clinic")
In this paper, we present the foundation of an mRNA-LNP platform for encoding and expressing therapeutic antibodies in vivo, eliminating the need for costly and time-consuming manufacturing.
