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.

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.

Proteomics Tools: A Resurrection

For decades, the evolution of proteomics has lagged. Now, a resurrection is underway. New tools are dismantling limitations, signifying a maturation of systems biology.

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.

Preventive Control Of Sequencing Through The Insert

Understanding library size distributions helps prevent sequencing through the insert, reducing adapter read‑through. Explore how smear analysis guides optimal read‑length selection to avoid noise.

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.

Structural Characterization Of RNA And Detection Of RNA-Ligand Binding

Delve into the intricacies of riboswitch behavior and ligand interactions and gain valuable insights into gene regulation and potential therapeutic targets.

Demystifying CRISPR gRNA Chemical Modifications

Chemical modifications on CRISPR gRNAs enhance stability, editing efficiency, and reduce immune responses, enabling in vivo applications and clinical success.

Pre-Banking And Avoiding Manufacturing Challenges For GMP Plasmids Containing Unstable Sequence Regions

Ensuring consistent replication and proper retention of plasmid DNA sequence is vital to the economics and efficiency in generating starting materials used for production of AAV delivered gene therapy and mRNA vaccines and therapeutics.

Biopharma Production And Quality Control With Electrophoresis

Discover how Kaneka Eurogentec, a leading provider of custom DNA synthesis and molecular biology services, enhanced their fragment analysis to achieve higher efficiency and accuracy in their workflows.