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
-
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.
-
Small, early operational decisions accumulate over time, creating hidden variability that emerges at scale, making standardization essential for stability, reproducibility, and regulatory confidence.
-
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.
-
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.
-
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.
-
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.
-
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.