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.

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.

Quality Control Of Oligonucleotides Using HPLC Coupled To UV And MS Detection

Efficient quality control of therapeutic oligonucleotides is crucial. Explore advanced methods combining HPLC-UV and mass spectrometry for precise impurity quantification and mass confirmation.

Accelerating IND-Enabling Studies With Automated Bioanalysis Workflows

Reimagined as automation-first, bioanalysis shifts from being an Investigational New Drug (IND) bottleneck to a catalyst for innovation—helping companies move therapies forward with greater confidence.

Pre-Banking And Avoiding Manufacturing Challenges For GMP Plasmids Containing Unstable Sequence Regions

Ensuring consistent replication and proper retention of plasmid DNA sequence is vital to the economics and efficiency in generating starting materials used for production of AAV delivered gene therapy and mRNA vaccines and therapeutics.

Optimizing qPCR For Broad-Range Transcript Detection In Anti-PD-1 Therapy

Optimize qPCR protocols for greater sensitivity and reproducibility to accurately measure immune-related gene expression and better identify patients who will benefit from anti-PD-1 immunotherapy.

Lentiviral Vector Characterization For Cell And Gene Therapy Manufacturing

This study focuses on characterizing recombinant LVVs used in gene-modified cell therapies, utilizing two real-time PCR assays adapted to the QuantStudio™ Absolute Q™ Digital PCR System.

Precision In Nanoparticle Processing And Monodispersed Lipid Nanoparticles For Advanced Therapeutics

Precision in nanoparticle processing is key for drug delivery and vaccines. Learn how monodispersed formulations enhance reproducibility and stability for next-generation therapeutics, like RNA-LNPs.