FDA's Platform Guidance Raises Long-Term Questions For mRNA Makers

As I read through the FDA's draft platform designation guidance and listened to various panel discussions the past few months, a handful of additional observations and considerations came to mind that are important to reiterate for the RNA space, particularly as we advance into therapeutics development.

Two "Spicy" Takeaways On The FDA's Platform Designation Guidance

Over the past few weeks since the guidance was released, I’ve spent some time listening to industry conversations and reading the guidance and related articles. In the following series of articles, I’ll share a few general impressions and (slightly spicy) thoughts I’ve been mulling over in response to the draft guidance. 

Developing & Manufacturing Oligonucleotides: An Overview

An overview of considerations for the design and development, quality control and characterization, regulatory submissions, and future directions for oligonucleotides.

Bridge RNAs: Principles, Applications, And Challenges

Bridge RNAs are structured noncoding RNAs that facilitate programmable recombination between target and donor DNA, enabling precise genomic rearrangements. A significant advantage is their modularity.

From "Up & Coming" To "Fully Formed": Maturing Our mRNA Outsourcing Partnerships

In the first- of this three-part article series, several mRNA execs share their perspectives on the world of partnering in the RNA therapeutics space, assessing first the current state of CDMO partnerships and how they anticipate this critical supportive infrastructure will grow in the years ahead.

A "Hot Take" On mRNA Critical Process Parameters

Bushwacking up a trailless mountain — as many of us may feel we’re doing today with the development of our mRNA therapeutics — is bound to be accompanied by its fair share of drama. These dynamics became increasingly apparent to me as I listened to a presentation unpacking the results of a BioPhorum benchmarking survey on CPPs for plasmid linearization, mRNA drug substance, and mRNA drug product production.

Expectations On The Pathway To GMP For Gene-Modified Cell Therapies

Learn how a robust understanding of the RNP’s critical process parameters and CQAs is crucial for ensuring product consistency, enhancing patient safety, and maintaining regulatory compliance.

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.

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.

Extending The Lower Limits Of Quantification Of A Therapeutic Oligonucleotide Through Microflow LC-MS/MS

Here a solution is presented to widely recognized challenges with ionpairing reversed phase liquid chromatography (IP-RP LC) through a novel microflow LC-MS strategy.

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.

Five Essentials For Accurate Oligonucleotide Chemistry

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

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.