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.

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.

From Data To Decisions: Accelerating Your mRNA Drug Development With Analytics

To stay ahead in the competitive landscape of mRNA development, adopt a comprehensive QC strategy today and begin conducting meaningful tests to ensure the quality of your test articles.

5 Key Questions On How To Accelerate mRNA Development

Stakeholders are working to solve key mRNA manufacturing challenges, including how raw materials impact product quality and how to address scale up. Though existing analytical and manufacturing technologies may fall short for mRNA, investment in innovative techniques will help meet increasing demand for personalized therapeutics.

The Value Of Peptones For Enhancing Biopharmaceutical Productivity

Peptones, versatile media additives derived from various sources, offer numerous benefits for biopharmaceutical production.

Gene Therapy And Oligonucleotide Compendium

Technical summaries and detailed protocols for purity testing, sizing, sequencing and expression analysis of oligonucleotide-based active agents as well as protein and lipid-based delivery systems.

Exploring Osmolality As A CQA Within RNA Therapeutics

Discover how osmolality can be applied throughout the bioprocessing workflow for RNA, and recognize the importance of this sensitive measurement, from formulation optimization to cryopreservation techniques.

CRISPR sgRNA For Successful Gene Editing: Discovery To Clinic

The single guide RNA (sgRNA) is one of the key components of successful CRISPR gene editing. Here we cover all the important aspects of sgRNA and Synthego’s sgRNA products.