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.

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.

Targeted LC-MS/MS Detection Of Lipid Impurities In Lipid Nanoparticles

Learn about an HPLC-MS/MS method for detecting lipid impurities in lipid nanoparticles (LNPs) and why it is critical for quality control of LNP-based drug delivery systems.

CRISPR Off-Target Editing: Prediction, Analysis, And More

A crucial consideration for researchers is off-target editing – unintended DNA modifications at sites other than the intended target. Explore the complexities and strategies to mitigate these effects.

Automated NGS Solutions Advance Rare Disease Research

See how a unified NGS workflow is helping a major pediatric genetics lab boost efficiency, manage growing sample volumes, and accelerate insights into rare diseases through streamlined automation.

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.

Exploring Osmolality As A CQA Within RNA Therapeutics

Discover how osmolality can be applied throughout the bioprocessing workflow for RNA, and recognize the importance of this sensitive measurement, from formulation optimization to cryopreservation techniques.

Top 8 Benefits Of Mass Spectrometry Imaging Early In Drug Development

Here we explore the top 8 benefits of using mass spectrometry imaging in the oncology drug discovery and early development stages.