Featured Editorial

81. In Vivo Genetic Medicines Need a Guide: The Answer Lies in Harnessing the Transcriptome 7/15/2024

    This article by Radar Therapeutics explains why the DNA/RNA revolution is underway and why solving specific targeting is next.

82. End-To-End mRNA DS And DP Manufacturing Processes 7/10/2024

    This article demystifies the manufacture of mRNA encapsulated in lipid nanoparticles across unit operations for plasmid linearization, drug substance (DS) manufacture, and drug product (DP) manufacture.

83. Innovation Advances In mRNA Medicine 7/9/2024

    The post-pandemic mRNA pipeline surge has led to a substantial investment in innovation. Let’s look at nonamplifying mRNA, saRNA, and circRNA.

84. Making Cancer Vaccines Is Complex; New Platform Guidance Could Help 7/3/2024

    FDA's draft guidance on platforms is mostly for established, well-characterized modalities, but it also has implications for the emerging area of personalized medicine.

85. CRISPR-Cas Genome Editing For Rejuvenation Of Aging Stem Cells 7/1/2024

    As stem cells age, their regenerative capacity declines due to accumulated genetic and epigenetic changes. Using CRISPR-Cas, scientists can directly edit these changes or modulate aging-related pathways.

86. A "Hot Take" on Personalized mRNA Medicines 6/20/2024

    mRNA’s small-scale and cell-free production are often-touted benefits in the personalized medicines sphere. But as I reviewed my notes from the BioPhorum ATMP member event, I found myself coming face to face with a difficult truth.

87. What Does FDA's Draft Platform Guidance Mean For CGT Companies? 6/17/2024

    Details of the FDA's highly-anticipated platform technology program were released in the form of a draft guidance. Let's explore with a former FDA/CBER regulator.

88. Do You Know Your QMM? Moderna Might Some Day 6/17/2024

    Don’t panic if you aren’t familiar with the above acronym, which stands for Quality Management Maturity. Chief Editor Louis Garguilo wasn't – until schooled by Jason Kerr, Regulatory Intelligence Senior Manager, Moderna and Adjunct Professor of Quality Systems, Keck Graduate Institute. QMM, it appears, may very well be in your future.

89. Have We Got Novel Drug Production And Compliance Timelines All Wrong? 6/13/2024

    Drug developers might want to consider longer-term approaches to meeting manufacturing and regulatory requirements.

90. A "Hot Take" On mRNA Scale-Up 6/12/2024

    In the following multi-part “mini-series” of RNA industry “Hot Takes,” I share several aspects that jumped out at me from the BioPhorum ATMP Event — starting with several takeaways about mRNA scale-up.