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.

Charged For Action: The Hidden Secrets Of AEX Oligonucleotide Analysis

Understanding how to fine-tune pH and ionic strength allows for the detection of truncated sequences and single-nucleotide variants, turning complex purification challenges into reproducible data.

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.

Alternatives To CRISPR-Cas9: Nucleases For Next-Gen Therapy

Alternatives to CRISPR-Cas9 address limitations like delivery challenges and off-target effects. Emerging nucleases offer improved specificity, smaller sizes, and therapeutic applications.

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.

The Hitchhiker´s Guide To Creating Oligonucleotides From Start To Finish

Producing high-quality oligonucleotides demands precise execution across multiple stages. Gain a clearer understanding of the complete mid-scale production workflow and necessary quality control measures.

Size Matters: Mastering Size Exclusion For Oligos

Learn how to separate molecules strictly by size, effectively remove salts, and select the correct pore specifications for a gentle, non-destructive purification workflow.

RNA Biotherapeutics — A New Approach To An Old Problem

Check out a brief history of RNA-based therapy and how Droplet Digital™ PCR supports this field of work.