Blog | November 20, 2023

ARW's CGT + RNA Manufacturing Must-Reads (TEACHER'S ADVICE EDITION)

Source: Cell & Gene Collaborative
ARW Edit Headshot 2

By Anna Rose Welch, Editorial & Community Director, Advancing RNA


Day-in and day-out, I write, read, listen to, and watch as much content as I can about CGT and RNA therapy manufacturing, in particular, and/or other ATMP industry-related topics that you should at least be aware of in the manufacturing facility. Every two weeks, I compile the articles and industry updates I think are most worthy of your time into an unconventional newsletter format (below) and send them out via email.

When this newsletter was sent, it was American Education Week, and Thursday, the 16th was specifically “Educator for a Day.” I could see no better way to embrace this theme than to showcase some of the whackiest pieces of advice teachers have had to say to their students — some of which can be loosely applied to the world of advanced therapy manufacturing. Keep misbehaving — but a friendly reminder to keep your face away from glue sticks. 

“Why is there a baby mouse up your sleeve? Please stop feeding him your pop tart.” 

“Unless there is someone teaching you how to read under that table, you need to come out and join us on the carpet.” 

  • I know — I, too, crave the darkness. But the light from the CGT policy space won’t burn us too badly. 
  • One of my favorite regulatory voices, Daniela Drago, teamed up with ASGCT’s Caitlin McCombs to lead “storypolicy time” this month, sharing their top five takeaways from ASGCT’s 5th Annual Policy Summit. You will need institutional access to read, however, so if you don’t have that, allow me to lower the tablecloth for you. 
  • OR you could stay out in the daylight and turn your eyes to the world of reimbursement policy, which I assure you is just as entertaining and delightfully dramatic as regulatory CMC policy. Here, this Cell & Gene article fills us in on some of the mechanisms certain states have put in place to facilitate greater access/reimbursement to CGTs, using the Sickle Cell Disease community as a prime example of what has and has not worked so far. 
  • In fact, the piece above nicely complements this feisty Wall Street Journal editorial chastising a proposed CMS rule that would require pharma companies to engage in a little game of “Show and Tell” and publish [unspecified] “confidential, proprietary, and competitively sensitive information” in a public forum to justify pricing and ensure Medicaid coverage. 

“How did you pull your tooth out with your shoelace while tying your shoe?” 

“Quit stuffing mulch down your pants.” 

  • Quantifying the amount of mulch that fits into a single pair of pants can be just as intimidating as quantifying a new modality’s patent landscape. 
  • Luckily when it comes to mRNA vaccine technology, the European Patent Office has put together this handy resource providing many facts, figures, and tables illustrating the growth of patenting in the infectious and oncological mRNA vaccine spaces, quantifying the number of international patent families, and identifying the most active patent applicants (companies & universities.) 
    • Worth noting, while mRNA patent filing obviously reached a peak in 2021, patent applications were on a steady clip upwards from 2015-2019, showcasing the growing interest/investment in mRNA pre-pandemic. 

“You did a great job putting on your belt. Now go back to the bathroom and turn your pants around.”

  • The big picture is hard. 
  • Thankfully, we now have an organization specifically focused on helping the mRNA industry keep its pants, shirts, and policies right-side-out/hemmed/tucked in, etc. The Alliance for mRNA Medicines launched in recent weeks, boasting an impressive lineup of 31 member companies and organizations. If you’re with an mRNA company or organization that wants to support the industry’s growth, check out the AMM site and apply for membership! 
  • Speaking of industry growth, the news out of Verve this week was fantastic: we now have positive Phase 1 data for mRNA-based in vivo gene editing (in the liver). The NYT did a profile of the trial and its results here
    • However, as I posted on LinkedIn, I was a bit disappointed that the term “mRNA” did not appear in the article at all. If you feel so inclined, comment on my post agreeing or disagreeing with my argument that mRNA should have made an appearance in the article. 

“This is not Star Wars… please stop pretending your fingers are ships.”

  • Talking about your AAV like it’s a space shuttle though? Carry on. 
  • In fact, Space Invaders may have come up in my Cell & Gene LIVE discussion with Regeneron’s Andrew Tustian and Regenxbio’s Justin Horvath on the analytical, upstream processing and downstream processing innovations taking shape in the AAV space to improve overall AAV quality. The full video of the hour-long discussion can be found here; shorter, more easily browsed segments are forthcoming! 
  • Many among us are also exploring how we can “trick out our ‘shuttles’” (i.e., explore novel capsids) to improve targeting, lower immunogenicity, and improve immune silencing. This LinkedIn post shares a full-text article on several leading methodologies being explored to improve discovery and selection of next-gen designer AAV capsids

“Please do not bring me presents from the toilet.”