Order, Chaos, And The mRNA Black Box: Takeaways From The USP/AMM Forum

To be clear, this year’s USP/AMM quality forum was just as focused on the intricacies of applying/using the methods. But what was equally if not more present in this year’s discussion was our increasing acknowledgement that our understanding of mRNA and its biological mechanisms remains quite messy.

From Supporting Component to Strategic Driver: The Evolving Role of gRNA in CRISPR Therapeutics

Guide RNA is evolving from a simple targeting reagent into a complex, engineered molecule shaped by expanding CRISPR modalities, increasing length and modification demands, and tighter integration with manufacturability, analytical control, and regulatory expectations.

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.

Stick With Me: The Cool Chemistry Of HILIC Oligonucleotide Analysis

Discover a powerful alternative for analyzing polar sequences without ion-pairing reagents. By leveraging hydrophilic partitioning, you can achieve sharper separation and improved mass spectrometry compatibility.

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.

Reduce Contamination Risks When Storing Synthetic DNA

An alternative to traditional polyethylene bags is offering cell and gene therapy manufacturers a solution to contamination concerns regarding DNA or RNA fragments.

Expectations On The Pathway To GMP For Gene-Modified Cell Therapies

Learn how a robust understanding of the RNP’s critical process parameters and CQAs is crucial for ensuring product consistency, enhancing patient safety, and maintaining regulatory compliance.

The Next Generation Of mRNA Products: Implications For Quality Control

To fully realize the potential of mRNA as a therapeutic or vaccine modality, developers will need to employ cutting-edge analytical methods to ensure the safety and efficacy of these novel products.

Elevating mRNA Manufacturing Toward GMP-Readiness

The mRNA Technology Transfer Program empowers LMICs with scalable vaccine production. Learn about a partnership that drives innovation in mRNA and sets a global blueprint for equitable health access.

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.