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.

Five Essentials For Accurate Oligonucleotide Chemistry

Explore five key areas in oligonucleotide chemistry where improvements will have a huge influence on your synthesis quality.

Preventive Control Of Sequencing Through The Insert

Understanding library size distributions helps prevent sequencing through the insert, reducing adapter read‑through. Explore how smear analysis guides optimal read‑length selection to avoid noise.

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.

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.

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.

Study Viral Infection In Resting Human CD4+ T Cells

Explore a study proving how synthetic single guide RNAs were used to optimize the editing conditions for resting human CD4+ T cells and maintain high cell viability for several weeks post Nucleofection.

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.