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
-
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.
-
Lipid nanoparticles enable efficient gene editing in hard-to-transfect cells like T-cells and blood stem cells, which offers scalable, clinically relevant methods for developing advanced therapies for cancer and rare diseases.
-
Batch processing platforms for LNPs lack efficiency, appropriate controls, and yield optimization. Continuous manufacturing platforms may address some of those issues.
-
Lipid nanoparticles have revolutionized drug delivery, ushering in a new era of nanomedicine. Discover how advanced analytical tools are accelerating next-generation LNP therapeutics.
-
Discover how an automated cryopreservation process is reshaping the leukapheresis supply chain and driving progress in the future of cell and gene therapy.
-
Producing high-quality oligonucleotides demands precise execution across multiple stages. Gain a clearer understanding of the complete mid-scale production workflow and necessary quality control measures.
-
Explore key considerations for manufacturing RNA-LNP drug products surrounding process and analytical development, automation, environmental monitoring, and strategic collaborations.