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.

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.

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.

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.

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.

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.

A&M STABTEST Expands Service Offerings In Protein And Oligonucleotide Analysis With LC-MS System

CRO upgrades systems to provide regulatory compliance for large-molecule protein analysis, expanding their oligonucleotides capabilities.

Data Processing For Mass Confirmation, Impurities Analysis Of Oligos

Explore an automated, compliance-ready LC-MS workflow for intact mass confirmation and purity analysis of synthetic oligonucleotides including siRNA and large single guide RNA oligonucleotides.

Gene Therapy And Oligonucleotide Compendium

Technical summaries and detailed protocols for purity testing, sizing, sequencing and expression analysis of oligonucleotide-based active agents as well as protein and lipid-based delivery systems.

Novel Plasmid DNA-Encoded Poly(A) Tails For mRNA Synthesis

Here, we evaluate the stability of these variant poly(A) regions and the biological activity of the mRNA containing the variant poly(A) tails made from these templates.

Exploring Osmolality As A CQA Within RNA Therapeutics

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.

Ultra-Sensitive Assay For Anti-Sense Oligonucleotide Quantification

Learn about a new level in sensitivity for challenging analytes in matrix that opens doors for accurate bioanalysis and metabolism studies with specificity not achievable with orthogonal methods.

mRNA Product Purification Process Strategies

Impurities from IVT can induce undesired immune responses and negatively impact translation efficiency. Explore the pros and cons of each option for mRNA purification.