Order, Chaos, And The mRNA Black Box: Takeaways From The USP/AMM Forum

To be clear, this year’s USP/AMM quality forum was just as focused on the intricacies of applying/using the methods. But what was equally if not more present in this year’s discussion was our increasing acknowledgement that our understanding of mRNA and its biological mechanisms remains quite messy.

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.

Sequencing Of Synthetic Oligonucleotides And Their Impurities

We demonstrate an automated, compliance-ready liquid chromatography-mass spectrometry (LC-MS) workflow for sequence confirmation of oligonucleotides and their impurities.

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.

NGS Analysis Of Hereditary Cancer Predispositions

Examine how a molecular genetics team improves NGS efficiency and library quality through streamlined automation, intuitive QC tools, and reliable workflows, boosting reproducibility.

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.

Determining RNA Integrity And Purity By Capillary Gel Electrophoresis

To ensure mRNA product quality, it's important to confirm the integrity of the RNA. Examine the results of a study challenging the performance of the CGE method in assessing mRNA integrity and purity.

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.

Advancing RNA-LNP Therapeutics As An Alternative To Viral Vectors

RNA-LNP therapeutics offer a flexible, transient alternative to traditional viral vectors for genetic medicine. Learn about the advantages of LNPs in safety, scalability, and payload versatility.