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.

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. 

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.

Limiting Off-Target Effects Of CRISPR-Based Products

Find out how to enhance CRISPR gene editing specificity by using Next Generation Sequencing to improve sgRNA quality control, addressing off-target effects and ensuring higher sequence fidelity.

IR Spectral Signatures Of GC, AU, And GU Base Pairing In H2O-Based Buffer By MMS

Explore a label-free approach to characterize RNA structure with high sensitivity. See how specific base pair types are resolved, providing a clearer picture of RNA folding.

Probing RNA Base Pairing And Ligand Interactions In Solution With Infrared-Based MMS

Discover why Microfluidic Modulation Spectroscopy (MMS) is a novel, label-free, solution-based infrared technique for probing RNA base pairing and ligand interactions.

Exploring The Spatial Distribution Of mRNA-Lipid Nanoparticles Using MALDI MSI

Learn how leveraging high-resolution MALDI-MSI can advance the development of lipid nanoparticle-based therapies, uncovering spatial biodistribution, pharmacokinetics, and organ-specific variability.

A Risk-Based Approach To Plasmid DNA And mRNA Process Development

Balancing robust analytics and clinical readiness is key for early-phase pDNA and mRNA therapeutics amid structural complexity and regulatory challenges.

Development Of Alternative BCDP Reagents For The Synthesis Of 5' -Terminal Phosphate Oligonucleotides

New alternative phosphitylating reagents reduce the safety hazards associated with BCDP, enabling a safer and effective synthesis of key oligonucleotide sequences.