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.

Expectations On The Pathway To GMP For Gene-Modified Cell Therapies

Learn how a robust understanding of the RNP’s critical process parameters and CQAs is crucial for ensuring product consistency, enhancing patient safety, and maintaining regulatory compliance.

Detection Of Adapter Dimers In NGS Libraries

Accurately identifying low‑level adapter dimers is key. Compare electrophoresis methods to show how different kits detect and resolve small fragments, helping researchers choose the best QC approach.

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.

Assigning IR Absorption Bands For RNA Building Blocks Using Microfluidic Modulation Spectroscopy

Explore the power of MMS in identifying RNA base signatures. This study analyzes vibrational modes of A, G, C, and U, revealing insights into electronic environments and molecular interactions.

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.

The Sleek Science Of Ion Pair (IP)-Reversed Phase (RP) Oligonucleotide Analysis

Master oligonucleotide separation by balancing hydrophobic and ionic interactions. Learn how selecting the right cationic pairing agent optimizes retention and ensures compatibility with mass spectrometry workflows.

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.