Blog | December 14, 2023


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.

For this final must-reads roundup of 2023, I figured I’d help us get into the holiday spirit by revitalizing the famous Christmas story — “A Visit from St. Nicholas” a.k.a. “Twas the Night Before Christmas”— to make it more applicable to the ATMP world. But should you already have your “CGT fanfiction” needs met elsewhere (somehow...), this batch of must-reads will, no doubt, provide way more value than my terrible rhyming.


‘Twas the night before Christmas and all through the house, four CEOs were stirring —

  • don’t quite know how they got into your house.
  • But, since it’s the season of polite cohabitation, perhaps you’ll be interested in hearing what they have to say about the past and future of the RNA therapeutics space? In my latest editorial, executives from Ethris, Replicate Biosciences, Combined Therapeutics, and Orbital Therapeutics share their visions of what’s to come both scientifically and commercially for RNA in the new year.
    • Please also note that this article is featured on one of Life Science Connect’s newest editorial communities, Advancing RNA, which (*spoiler alert*), is set to become my new editorial home in 2024. Stay tuned for more information in the new year!

You would’ve been tucked all snug in your bed, but out on the market there rose such a clatter,

When what to your wandering eyes should appear, but Peter Marks — with a beard! —

Marks whistled and shouted and called each platform by name:

  • On AAVs, LNPs, LVVs, Go! On cell therapy, gene therapy, in & ex-vivo, let’s roam!
  • A few weeks ago, ARM & NIIMBL hosted a scientific exchange meeting between the industry and FDA on “Building Blocks & Platform Development for CGT Development.” In case you couldn’t nerd out for the whole day (or at all), ARM released this helpful summary sheet sharing high-level information about the FDA’s platform technology provision and the scope of the scientific exchange meeting.
    • A much more comprehensive whitepaper is forthcoming, in which we’ll become privy to the specific building blocks each working group outlined for AAV, IPSC, and LNP technologies respectively.

And then, in a twinkling, down the chimney there came a pair of Marks’ Merrell footwear

He spoke not a word but went straight to his work, unloading gift after gift —

  • No supply chain impediments to drive him berserk.
  • If visions of sugar plums dancing in your head is getting dull, perhaps you can substitute in single-use systems and modular technologies. You won’t be alone; as a recent editorial from BioPlan Associates explains, interest in modular bioprocessing units/modular facilities remains evergreen — particularly in the personalized medicine sector.
  • President Biden just announced two big supply-chain-centric initiatives:
    • The new cabinet-level Council on Supply Chain Resilience will review the state of the U.S. supply chain to identify products (per sector) that are critical to national and economic security.
    • As per the Defense Production Act, the White House/HHS will be investing in the domestic production of starting materials for essential sterile injectable medicines.
  • As your favorite Hallmark movie cuts to commercial, perhaps turn your attention (3 minutes at a time) to this quick, 30-minute presentation, during which an executive at Roche delves into a few supply chain strategies the company has implemented to better control ATMP raw material COGS and to streamline personalized medicines’ delivery to clinics. (BTW, Roche has a very heterogenous pipeline of in- and ex-vivo viral vector-based AND mRNA therapies.)

And then up the chimney that right jolly regulator rose,

  • Was he real or just a hologram — oh dear, will you ever know?
  • Though resources on AI/ML can leave the non-digital experts among us feeling like we drank a bit too much spiked eggnog, I still appreciate this (Nov. 2022) no-nonsense article from McKinsey on how AI can accelerate R&D for CGTs. In particular, the authors walk us through the specific use-cases for AI in the R&D for mRNA cancer vaccines, CAR-Ts, and AAV gene therapies.
  • Though my partner who attended the AWS re:Invent Keynote did not manage to secure me an introduction to Pfizer’s Chief Digital & Technology Officer after her keynote, I appreciated this quick (albeit rah-rah Amazon!-heavy) write-up of takeaways from her presentation on Pfizer’s data management and AI strategy. A video of her keynote is available here from the 1:09:30-1:19:00 mark.

But you heard him exclaim, ere he hiked out of sight: “The highest quality therapies for all —