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.
This newsletter was sent on National Storytelling Day. So, I’ve prefaced each section of must-reads with some of the best opening lines of novels I’ve ever encountered, and I’ve taken the liberty of authoring the second line to prove that every book in the world could be written about ATMPs (but thankfully isn’t...). Happy reading!
‘Where’s Papa going with that axe,’ said Fern to her mother as they were setting the table for breakfast.
~ E.B. White, Charlotte’s Web
- “Papa’s CDMO promised something it couldn’t deliver,” Mama replied, cracking an egg into the frying pan.
- To get a practical look at the current state of the CGT CDMO outsourcing space (sans any axe-wielding angst), look no farther than the first of this three-part article series featuring two of my favorite ATMP outsourcing gurus, Adam Haskett & Joe Graskemper. Here, Haskett and Graskemper identify where the CDMO landscape has grown and in which areas it has yet to demonstrate greater capability.
They say the world is flat and supported on the back of four elephants who themselves stand on the back of a giant turtle. ~ Terry Pratchett, The Fifth Elephant
- Often overlooked is that the turtle — the base of all worldly balance — is leashed to the world’s most esteemed regulatory authorities.
- To ensure that your programs — and the entire ATMP development world — remain in balance, please meet with the FDA as often as you possibly can. In fact, the FDA just revised its 2017 guidance on formal meetings to include a few key updates. You can check out the guidance and a brief breakdown of the new additions in this LinkedIn post.
- If you’re feeling particularly chatty and/or inquisitive to the point that basic meetings aren’t enough, the FDA has just the thing for you! Meet the Support for clinical Trials Advancing rare Disease Therapeutics (START) pilot program. Lucky sponsors will be able to communicate more frequently on an ad hoc basis about a variety of development challenges.
- Because nothing causes the world to turn upside down more regularly than inefficient standard operating policies and procedures, the FDA’s CBER has been doing all it can to ensure its staff are up to speed with internal administrative processes for clinical holds, BLA filings, review periods…and the list goes on. RAPS details all the revisions being made in this article.
In the beginning, the universe was created. This made a lot of people very angry and has been widely regarded as a bad move. ~ Douglas Adams, The Restaurant at the End of the Universe
[INDUSTRY HOT TAKES]
For the better part of my childhood, my professional aspirations were simple — I wanted to be an intergalactic princess. ~ Janet Evanovich, Seven Up
- Little did I know that I’d grow up to be the Queen of CGT.
- As you cease your slow clap (thank you, thank you), I’d like to say that you, too, can feel like an intergalactic ruler of all CGT wisdom. There have been several market insight reports released in the past few weeks that are chock-full of riches. In particular:
- Deloitte released its 2023 CGT Industry Survey, which is free to download. Though manufacturing has its own dedicated section, manufacturing is referenced throughout the entire report, even in the functional areas you might least expect to find them.
- In recent weeks, Deloitte also hosted its Next-Gen Therapy Industry Working Group meeting, several takeaways from which can be found in this pithy LinkedIn article. (They’re all insightful, but #2 and #6 caught my eye in particular.)
- For those striving to better understand the state of current cell therapy analytical and manufacturing platform/technology offerings, CRO/CDMO services, and auto/allo COGS assessments, Deci Bio lays all this out for us in their newly released Cell Therapy R&D & Manufacturing Global Market Report 2023-2028 (for purchase only).
It was the day my grandmother exploded. ~ Iain Banks, The Crow Road
- I did not have a risk management plan in place to keep this turn of events at bay. Mom was pissed.
- Though this article was written a little too late to save Grandma, it’s hopefully not too late to revisit and/or improve your knowledge on the risk management tools offered within the recently revised ICH Q9 (R1). Here, two ValSource consultants break down the risk tools available to you and in which situations they may be most valuable to employ.
As Gregor Samsa awoke one morning from uneasy dreams, he found himself transformed in his bed into a gigantic insect. ~ Franz Kafka The Metamorphosis, and other stories
- “God#$%@,” he said, cursing what he could only assume was a grievous endogenous mRNA coding error.
- For the rest of us who aren’t living in an episode of The X-Files, here are a few reviews and journal articles published highlighting the myriad ways synthetic RNA is being explored/advanced therapeutically in non-sci-fi ways:
How five crows managed to lift a twenty-pound baby boy into the air was beyond Prue, but that was certainly the least of her worries. ~ Colin Meloy, Wild Wood
- She now had a giant baby nested on her porch, and she had no idea what to do with it.
- If you feel as overwhelmed as Prue, you might be interested in some “molecule parenting” classes from NIST. From November 1-3, NIST will be hosting three in-person and hybrid workshops discussing measurement needs for viral & nonviral gene delivery and current/future projects via the NIST Genome Editing Consortium and the NIST Flow Cytometry Standards Consortium.
- If that’s not epic enough for you, FDA, Standards Coordinating Body, NIST, and USP will be co-hosting a hybrid workshop Nov. 16-17 on standardization needs in cell and gene therapy development.
- Looking ahead into 2024, this virtual USP forum on mRNA vaccines and therapeutics is worth marking down for February 28-29. In fact, if you have any work you’d like to share at this forum, USP is currently seeking abstracts through the end of October.