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. 

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.

Quality Assurance In IVT RNA Vaccine Development Using Electrophoresis

Discover advanced solutions for IVT RNA synthesis, focusing on enhancing fidelity in throughput, innovative technologies, and methodologies to optimize your RNA research and development processes.

Mastering mRNA Manufacturing: A Focus on Raw Materials & Supply Assurance

Rapid advancements in mRNA-based therapeutic development have underscored the role of raw material quality and supply chain management in ensuring their successful production.

CRISPR sgRNA For Successful Gene Editing: Discovery To Clinic

The single guide RNA (sgRNA) is one of the key components of successful CRISPR gene editing. Here we cover all the important aspects of sgRNA and Synthego’s sgRNA products.

Why Bioanalysis Needs To Break Free From Manual Bottlenecks

Bioanalysis remains one of the most persistent bottlenecks in drug development, with CROs displaying inefficiencies that are no longer operational nuisances but strategic liabilities.

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.

High-Throughput Characterization Of RNA

How a multi-capillary electrophoresis (CE) system is used to efficiently separate a wide range of RNA-based molecules by size and demonstrates the multiplex capability for RNA characterization.