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.

How A Drug's Molecule Type Affects Several Bioanalysis Parameters

Discover why bioanalytics are crucial to selecting promising drug candidates during development and to characterizing the pharmacokinetic and pharmacodynamic profile of the drug in early clinical trials.

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.

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.

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.

Preventive Control Of Sequencing Through The Insert

Understanding library size distributions helps prevent sequencing through the insert, reducing adapter read‑through. Explore how smear analysis guides optimal read‑length selection to avoid noise.

The Future Of Small And Large Molecule Bioanalytics

Learn how innovative, emerging technologies are revolutionizing the field of bioanalytics and drug development.

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.