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. 

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.

Unlocking The IP Benefits Of Novel Nucleases In CRISPR Therapies

Incorporating novel nucleases into CRISPR therapies enhances intellectual property benefits through improved licensing, strategic protections, and access to new gene targets.

Understanding The Intricacies Of qMSI

Explore quantitative mass spectrometry imaging (QMSI) and leading spatial bioanalysis and biology services that offers one-on-one support throughout your drug development process.

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.

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.

Novel Plasmid DNA-Encoded Poly(A) Tails For mRNA Synthesis

Here, we evaluate the stability of these variant poly(A) regions and the biological activity of the mRNA containing the variant poly(A) tails made from these templates.

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.