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.

Alternatives To CRISPR-Cas9: Nucleases For Next-Gen Therapy

Alternatives to CRISPR-Cas9 address limitations like delivery challenges and off-target effects. Emerging nucleases offer improved specificity, smaller sizes, and therapeutic applications.

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.

Selection And Evaluation Of Hybridization Capture Probes For LC/MS Analysis Of Oligonucleotides

To overcome challenges in oligonucleotide LC/MS bioanalysis, consider optimizing probe design by evaluating alternative chemistries like PNA for improved recovery and chromatographic performance.

Exploring The Spatial Distribution Of mRNA-Lipid Nanoparticles Using MALDI MSI

Learn how leveraging high-resolution MALDI-MSI can advance the development of lipid nanoparticle-based therapies, uncovering spatial biodistribution, pharmacokinetics, and organ-specific variability.

Lentiviral Vector Characterization For Cell And Gene Therapy Manufacturing

This study focuses on characterizing recombinant LVVs used in gene-modified cell therapies, utilizing two real-time PCR assays adapted to the QuantStudio™ Absolute Q™ Digital PCR System.

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.

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.