FORMULATION ARTICLES
-
Why Delivery Is Defining The Next Era Of Neuromuscular Therapeutics
Emerging 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.
-
Analytical Platforms: The Foundation Of The Next Generation Of RNA Therapeutics
RNA therapeutics are entering a new era where analytical platforms drive scalability, regulatory confidence, manufacturing flexibility, and the development of increasingly complex products.
-
A New Purification Strategy For The Removal Of dsRNA Impurities From IVT mRNA Products
NIST 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.
-
The Second Holy Grail Of Gene Editing: Large Knock-Ins
Exploring the race to achieve large gene knock-ins, emerging editing platforms, and how overcoming this challenge could unlock therapies for many inherited diseases.
-
From Baby KJ To Global Access: The 3 Pillars Of Next-Gen mRNA Expansion
Obviously, 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.
-
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.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
-
Messenger RNA-LNPs are a promising strategy for delivering gene-editing components. Learn how a continuous manufacturing platform can address the scalability and reproducibility challenges of this process.
-
Well-designed lipid nanoparticles enable safe, efficient delivery. OVA-based models show how formulation choices shape efficacy, safety, and anti-tumor outcomes in melanoma research.
-
Learn about the key stages of drug discovery and screening as well as how to overcome bottlenecks in screening mRNA-LNPs to rapidly develop genomic medicines.
-
Mastering IVT yield requires balancing magnesium and nucleotide ratios. Learn how specific buffers and pyrophosphatase optimize mRNA synthesis for scalable, high-quality therapeutic production.
-
From fermentation to formulation, mRNA manufacturing requires a toolbox of technologies, services, and expertise. Explore our portfolio of solutions for making, purifying, and formulating mRNA.
-
RNA therapeutics hold immense promise for precision treatment, but their development faces challenges. A CDMO with adaptable platforms and flexible timelines can help reduce time and costs.
-
Fast-track RNA-LNP development, streamline screening with off-the-shelf ionizable lipid mixes, and utilize validation strategies to generate reproducible and scalable LNPs for lead candidates.