In 1998, the US Food and Drug Administration (FDA) approved fomivirsen as the first therapeutic oligonucleotide therapeutic. This approval marked a revolution of mechanism of action discovered decades before finally coming to fruition. Since then, the landscape of chemical modifications of oligonucleotides, conjugations and formulations has evolved tremendously, contributing to improvements in stability, efficacy and safety. Today, more than a dozen antisense oligonucleotides (ASOs) and small interfering RNA (siRNA) drugs are on the market, most of which are designated as orphan drugs for treating rare genetic diseases.
Biopharma
Biologics characterization pose many challenges due to the diversity of the biomolecules. Learn about the many powerful tools that allow researchers to fully characterize and quantify their biologic drugs, using mass spectrometry and capillary electrophoresis instrumentation.
Supporting new CRISPR gene editing systems
Prime editing (PE) is a next-generation gene editing technology that utilizes a Cas9 protein fused to a prime editing guide ribonucleic acid (pegRNA) to achieve high CRISPR/Cas9 editing efficiency and precision. However, the length requirement of pegRNAs at 120–250 nucleotides (nt) and their high level of secondary structure formation present analytical challenges for the purity analysis of chemically synthesized pegRNAs during development and quality control (QC).
A new approach for comprehensive AAV evaluation including full and empty analysis
Certain next-gen vaccines and gene therapy applications rely on the usage of adeno-associated viruses (AAV) as a delivery vehicle. To ensure the safety and efficacy of viral vector drugs, multiple critical quality attributes (CQAs) need to be well characterized.
Guide decisions during cell line development with more information at the intact level
Monitoring product quality attributes (PQAs) throughout monoclonal antibody (mAb) development is vital to ensuring drug safety and efficacy. By adopting orthogonal analytical techniques and integrating new technologies that have the potential to provide more information, it is possible to improve product quality and manufacturing efficiency and make more informed decisions.
Better mRNA-LNPs: encapsulation efficiency, mRNA integrity and purity, lipid N-oxides and beyond
Lipid nanoparticles (LNPs) are widely used vehicles for mRNA-based therapeutics and vaccines. However, ionizable lipids used in LNPs can be susceptible to N-oxide impurities that can cause functional loss of the mRNA cargo.
Adducts and N-oxides: understanding lipid nanoparticles (LNPs) for better mRNA drugs
Lipid nanoparticles (LNPs) are widely used vehicles for messenger RNA (mRNA)-based therapeutics and vaccines. However, ionizable lipids used in LNPs can be susceptible to N-oxide impurities that can cause functional loss of the mRNA cargo.
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The costly consequences of unplanned downtime
Unplanned downtime is a formidable adversary that businesses across various industries strive to minimize. Defined as the unexpected interruption of regular operations, unplanned downtime can wreak havoc on productivity, profitability and customer satisfaction. In this article, we delve into the causes of unplanned downtime, its far-reaching consequences and strategies to mitigate its impact.
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Beyond mRNA: how advances in analytical techniques are enabling a revolution in the RNA drug landscape
Currently, there are 3 main types of in vitro transcribed (IVT) RNA drugs. Two of these—conventional messenger RNA (mRNA) and base-modified mRNA (bmRNA), which incorporates chemically modified nucleotides—are non-replicating. The third type is self-replicating RNA (srRNA), which is based on an engineered viral genome but devoid of viral structural protein genes. Its self-replicating ability makes srRNA a promising tool for new therapeutic drugs.
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Changing the game of drug development with the Intabio ZT system
What better to learn how a new technology like icIEF-UV/MS is advancing drug development than hearing from the labs that are using it?
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The whys behind the dos and don’ts of oligonucleotide analysis
We know that LC-MS oligonucleotide analysis can have its share of challenges—challenges with sensitivity, challenges with adduct formation and challenges with data analysis, to name just a few. That’s why this blog takes a closer look at the dos and don’ts of this type of analysis and explores some keys to success. It also explains why following these simple rules can vastly improve your oligonucleotide characterization and quantitation efficiency and success.