ANALYTICAL/QUALITY ARTICLES
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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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5 Key Questions On How To Accelerate mRNA Development
Stakeholders are working to solve key mRNA manufacturing challenges, including how raw materials impact product quality and how to address scale up. Though existing analytical and manufacturing technologies may fall short for mRNA, investment in innovative techniques will help meet increasing demand for personalized therapeutics.
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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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The Absolute Essentials Of Manufacturability
Learn why integrating Quality by Design (QbD) and Process Analytical Technology (PAT) early on is essential for developing a robust, controlled, and financially viable process.
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Effects Of mRNA Capping Technologies
This study compares two important mRNA capping technologies by evaluating the mRNA integrity, percent Cap1 content, dsRNA content, in vivo expression, and innate immune activation in mice.
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Quantification Of Large Oligonucleotides Using High Resolution MS/MS
Explore how to achieve sensitive, high-resolution analysis of large oligonucleotides, with the opportunity to perform both qualitative and quantitative analysis in a single run.
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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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CRISPR sgRNA For Successful Gene Editing: Discovery To Clinic
The single guide RNA (sgRNA) is one of the key components of successful CRISPR gene editing. Here we cover all the important aspects of sgRNA and Synthego’s sgRNA products.
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The Value Of Peptones For Enhancing Biopharmaceutical Productivity
Peptones, versatile media additives derived from various sources, offer numerous benefits for biopharmaceutical production.
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Reduce Contamination Risks When Storing Synthetic DNA
An alternative to traditional polyethylene bags is offering cell and gene therapy manufacturers a solution to contamination concerns regarding DNA or RNA fragments.