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.

Optimizing mRNA For Success: A Guide To Enhanced Stability, Delivery, And Efficacy

RNA offers exciting potential for vaccines, gene therapies, and personalized medicine, but challenges related to stability, delivery, and manufacturing persist.

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.

How To Navigate Through The Jungle Of Columns?

Whether prioritizing mass spectrometry compatibility or loading capacity, understanding the specific trade-offs between IP-RP, AEX, and SEC methodologies is essential for successful characterization.

Size Matters: Mastering Size Exclusion For Oligos

Learn how to separate molecules strictly by size, effectively remove salts, and select the correct pore specifications for a gentle, non-destructive purification workflow.

Optimizing qPCR For Broad-Range Transcript Detection In Anti-PD-1 Therapy

Optimize qPCR protocols for greater sensitivity and reproducibility to accurately measure immune-related gene expression and better identify patients who will benefit from anti-PD-1 immunotherapy.

Study Viral Infection In Resting Human CD4+ T Cells

Explore a study proving how synthetic single guide RNAs were used to optimize the editing conditions for resting human CD4+ T cells and maintain high cell viability for several weeks post Nucleofection.

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.