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.

The "Middle Ground" Advantage: Why Alnylam Is Betting Big On Chemoenzymatic Ligation

Though conversations are picking up around this hybrid approach, we aren’t exactly known as an industry that embraces risk no-holds barred. While there are a handful of manufacturers and CDMOs who are embracing the hybrid model today, there are many more that are likely to hold out for the next-next gen approach: Fully enzymatic oligo production. However, as Alynlam’s CTO and Chief Quality Officer Tim Maines argued, embarking into the “middle ground” of enzymatic ligation is essential for garnering the step-by-step learnings needed to help us unlock even bigger manufacturing wins in the future. 

The Monster Under The Bed: Defining Potency in the mRNA Space

During the CASSS mRNA Symposium last spring, a seemingly simple, straightforward question was raised: How are we defining and measuring our product’s potency? But as we already know, this is far from a straightforward question — and, dear reader, based on the resulting discussion amongst regulators and panel members, it became clear we have a lot of different definitions on what potency even is in the mRNA space.

Innovations In The Oligonucleotide Supply Chain: Regulatory Considerations For Materials, Manufacturing, And Lifecycle Control

Oligonucleotide therapeutics have rapidly advanced into late-stage and commercial development, shifting regulatory focus toward the maturity of manufacturing and supply chain control rather than therapeutic novelty. Regulatory success now depends on how effectively sponsors translate innovative chemistries into well-characterized, scalable, and sustainable materials and processes.

Advancing Genomic Discoveries With Comprehensive Sequencing Support

See how a leading genomics facility advances long‑read sequencing through rigorous QC and flexible workflows, offering practical insights for researchers aiming to improve sequencing outcomes.

Oligonucleotides: Where We Are And Where We Would Like To Go 

This presentation provides an overview of oligonucleotide bioanalysis's current status and future directions. We will look into the advantages and disadvantages of various bioanalytical techniques.

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.

Nanomedicine Modalities Find Path To Clinic Through Analytics

Lipid nanoparticles have revolutionized drug delivery, ushering in a new era of nanomedicine. Discover how advanced analytical tools are accelerating next-generation LNP therapeutics.

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.

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.

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.