FORMULATION ARTICLES

• FDA Issues New Guidance: Nonclinical Safety Assessment Of Oligonucleotide-Based Therapeutics

  FDA's CDER recently issued a draft guidance: Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics. This article provides a summary and analysis. The public comment period ends January 14, 2025.

• Commercializing mRNA: 3 "Action Items" To Turn Promising Science Into Real-World Impact

  The recent Alliance for mRNA Medicines’ inaugural Ascent conference provided us with a lot of great updates on the mRNA industry’s progress. But throughout each of these specific conversations, there was a shared underlying question: How can we become a less esoteric industry?

• A "Flight Plan" For Achieving These 3 mRNA Development Milestones

  Here, in the final installment of this three-part series, I’ll share the panelists’ greatest learning experiences and regulatory advice (so far) on what we should be looking out for as we strive to achieve three big milestones: Entering the clinic; implementing advanced manufacturing technologies into our processes; and standardizing our manufacturing platforms.

• Translatability: The Challenge Keeping mRNA Therapeutics Execs Up At Night

  It goes without saying that there’s a lot we need to understand about how our drug products’ composition (i.e., lipid/mRNA ratio) analytical profile, and manufacturing process impact their potency and safety. But I also appreciated the panels’ focus on the importance of gaining a clearer understanding of the biological factors that influence the strength, durability, immunogenicity, and overall deliverability of our RNA therapeutics in vivo.

• Collaboration Key To Going "Out On A Limb" For Nonviral mRNA Delivery

  As the members of this panel at the World Vaccine Congress emphasized, they see great — and in some cases untapped — opportunities for collaboration to better advance nonviral delivery of a wide variety of RNA therapeutics. 

• Two "Hot Takes" On mRNA Delivery

  As I argued in a previous “Hot Take,” “the devil is in the details” when it comes to developing both our drug substances and our drug products. We need to get to the point where we have more details and knowledge about alternative methods of delivery (beyond LNPs) so we can make educated decisions on the best path forward for delivering our products.