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.

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.

Engineered Enzymes Powering The Future Of mRNA Therapeutics

Optimizing RNA polymerase selection significantly improves mRNA yield and purity while reducing dsRNA. Discover how advanced enzyme engineering enhances therapeutic manufacturing efficiency and safety.

Learn How Compliance-Ready LC-MS Workflows for Oligonucleotides Can Expand your Analytical Services Portfolio

CMO invests in solution to ensure data integrity and meet regulatory requirements as they expand their oligonucleotide manufacturing and analysis services.

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.

Gene Therapy And Oligonucleotide Compendium

Technical summaries and detailed protocols for purity testing, sizing, sequencing and expression analysis of oligonucleotide-based active agents as well as protein and lipid-based delivery systems.

Establishing Analytical Methods For mRNA-Based Therapies

Here, we provide a detailed description of assays for sequence identification and LNP composition in mRNA-LNP products that support the development of safe and effective mRNA therapies.

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.