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.

Best Practices For Analysis Of In Vitro Transcribed (IVT) mRNA

Learn best practices for high-resolution analysis of IVT RNA using automated capillary electrophoresis. Improve accuracy and precision in quantifying critical quality attributes.

Data Processing For Mass Confirmation, Impurities Analysis Of Oligos

Explore an automated, compliance-ready LC-MS workflow for intact mass confirmation and purity analysis of synthetic oligonucleotides including siRNA and large single guide RNA oligonucleotides.

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.

Metabolite ID And Relative Quantification Of Oligonucleotides In Plasma

This technical note describes the identification, relative quantification, and structural confirmation of the chain-shortened metabolites of a phosphorothioated oligonucleotide.

Determining RNA Integrity And Purity By Capillary Gel Electrophoresis

To ensure mRNA product quality, it's important to confirm the integrity of the RNA. Examine the results of a study challenging the performance of the CGE method in assessing mRNA integrity and purity.

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.

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.