ANALYTICAL/QUALITY ARTICLES
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Order, Chaos, And The mRNA Black Box: Takeaways From The USP/AMM Forum
To be clear, this year’s USP/AMM quality forum was just as focused on the intricacies of applying/using the methods. But what was equally if not more present in this year’s discussion was our increasing acknowledgement that our understanding of mRNA and its biological mechanisms remains quite messy.
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From Supporting Component to Strategic Driver: The Evolving Role of gRNA in CRISPR Therapeutics
Guide RNA is evolving from a simple targeting reagent into a complex, engineered molecule shaped by expanding CRISPR modalities, increasing length and modification demands, and tighter integration with manufacturability, analytical control, and regulatory expectations.
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The RNA Ties That Bind: 3 Takeaways From The OPT Congress
Here, I unpack three takeaways I had from the OPT Congress, each of which I’d argue are indicative of the interconnectedness across the genetic medicines field.
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Rethinking ATMP Assurance When Sterility Timelines, Reality Misalign
The compendial 14-day sterility testing window exposes product and patient to numerous risks, but settling on a rapid alternative also presents its own set of challenges.
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MicroRNA Therapeutics In Metabolic Liver Disease: Context-Driven Drug Discovery
Realizing the therapeutic potential of miRNAs in liver disease requires confronting a problem the field has not fully reckoned with.
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Inside Alnylam's Playbook For High Volume siRNA Production
As one of the first companies to establish a chemoenzymatic ligation platform, Alnylam's Maines and Nechev are in the perfect position to espouse wisdom on the practical aspects and remaining challenges of implementing enzymatic ligation at a large scale. Here in this installment, Maines and Nechev outline what they’ve learned thus far about the science of enzymatic ligation, while also paying credence to the unknowns and existing barriers that, as a frontrunner, Alnylam will inevitably (but willingly) be tasked with facing in the future.
ARTICLES, APP NOTES, CASE STUDIES, & WHITE PAPERS
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Understanding The Intricacies Of qMSI
Explore quantitative mass spectrometry imaging (QMSI) and leading spatial bioanalysis and biology services that offers one-on-one support throughout your drug development process.
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RNA Biotherapeutics — A New Approach To An Old Problem
Check out a brief history of RNA-based therapy and how Droplet Digital™ PCR supports this field of work.
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Comparison Of Constant- And PulsedField Electrophoresis
Accurate sizing of large DNA fragments is crucial in long‑read sequencing. Examine how different electrophoresis methods perform with 10–20 kb smears and how to choose the most reliable approach.
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Twist Of Two: 2D-LC For Oligos
Discover how coupling orthogonal mechanisms reveals hidden impurities and creates a more robust strategy for characterizing complex oligonucleotide samples.
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CRISPR Off-Target Editing: Prediction, Analysis, And More
A crucial consideration for researchers is off-target editing – unintended DNA modifications at sites other than the intended target. Explore the complexities and strategies to mitigate these effects.
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Metabolite ID And Relative Quantification Of Oligonucleotides In Plasma
This technical note describes the identification, relative quantification, and structural confirmation of the chain-shortened metabolites of a phosphorothioated oligonucleotide.
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Methods for mRNA Poly(A) Sizing
This study used Oxford Nanopore Technologies' Direct RNA Sequencing Kit and obtained successful sizing of poly(A) tail regions. The results are presented in a histogram and table format.