READ: ARW ON RNA

1. Shifting Sands Scales: Manufacturing Considerations For Small-Scale mRNA Production 9/30/2024

   Though mRNA may, in theory, be the “perfect technology” for scaling up/out and down, several industry experts from a panel during Cytiva’s recent RNA-LNP Summit explained that the successful transition from large to small-scale, high-quality mRNA production demands specific manufacturing capability improvements/advancements. In the first of this two-part article, I outline a few of these important manufacturing and outsourcing considerations that must accompany our shift to smaller scales.

2. One Unspoken Issue With The FDA's Platform Designation 9/19/2024

   Subjectivity and hearsay will no doubt play a big role in how we approach the FDA's Platform Designation guidance moving forward, and I daresay these hard to mitigate factors will complicate our relationship with this designation in the future. 

3. FDA's Platform Guidance Raises Long-Term Questions For mRNA Makers 9/5/2024

   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.

4. Two "Spicy" Takeaways On The FDA's Platform Designation Guidance 8/29/2024

   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articles, I’ll share a few general impressions and (slightly spicy) thoughts I’ve been mulling over in response to the draft guidance. 

5. Is This The Hardest Question Facing mRNA Therapeutics Makers? 8/18/2024

   As I’ve been reminded on a few different occasions, there is a difficult truth we may need to wrap our brains around in certain situations: Just because we can make an mRNA therapy for an indication, doesn’t necessary mean we should — or that the patient population will necessarily want it or be able to access it. 

6. Translatability: The Challenge Keeping mRNA Therapeutics Execs Up At Night 8/9/2024

   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.

7. Collaboration Key To Going "Out On A Limb" For Nonviral mRNA Delivery 7/31/2024

   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. 

8. From "Up & Coming" To "Fully Formed": Maturing Our mRNA Outsourcing Partnerships 7/24/2024

   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.

9. A "Hot Take" On mRNA Critical Process Parameters 7/17/2024

   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.

10. Two "Hot Takes" On mRNA Delivery 7/2/2024

    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.