Blog | October 23, 2023

ARW's CGT+RNA Manufacturing Must-Reads (ARW IS ON A DEADLINE Edition!)

Source: Cell & Gene Collaborative
ARW Edit Headshot 2

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

missed opportunity, deadline, urgent time GettyImages-1412793246

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 out, I was on a deadline for a forthcoming Life Science Leader magazine article. So, in honor of my colleague who (nicely but quite directly) said he’d kill me if I miss said deadline, I introduced each section of these must-reads with a reference to some of our favorite “speedy” cartoon/comic book characters — just to keep things moving full speed ahead. God speed.

All of us have powers, skills, and abilities. Where I come from, heroes use those powers to...make the world a better place, whether it’s their world or not. ~ The Flash

Andale! Andale! Arriba! Arriba! Yii-ha! ~Speedy Gonzalez

  • Regulators are hardly the speediest mice in all the land. But they’re poised to make some big contributions to the CGT/RNA spaces in the months/years ahead.
  • For starters, the FDA will be hosting an advisory committee meeting on October 31 for Vertex/CRISPR Therapeutics’ exa-cel, an ex vivo, CRISPR-edited gene therapy.
    • For a good write-up on exa-cel’s clinical results and safety concerns (as of June 2023), check out this article.
  • Across the pond, the EMA is hard at work putting together guidelines to address the unique complexities of oligonucleotide development, manufacturing, and quality. This article provides a great breakdown of the anticipated scope of the guidelines, which are being crafted following industry commentary on a September 2022 concept paper.

Warning! The Tasmanian Devil is a powerful, vicious, evil-tempered brute — hungry at all times! It will eat anything but is especially fond of...

I’m the top banana in a world full of hungry monkeys. ~Sonic the Hedgehog

  • Intellia to Sonic: “Hold my peel.”
  • We’ve been starving for late-stage clinical data demonstrating mRNA’s therapeutic (as opposed to prophylactic) potential, and now, thanks to Intellia, we’re on the verge of getting some. Intellia received FDA clearance to begin a Phase 3 trial of its in vivo (mRNA-based) CRISPR gene editing therapy.
  • In 11 days, the mRNA industry will welcome its dedicated trade group, Alliance for mRNA Medicines. The Alliance previously held a “soft launch” at BIO in June and is poised to officially launch in Berlin at the mRNA Healthcare Conference.
  • I’ve spent the past few weeks interviewing 4 executives for my forthcoming 2024 RNA Outlook article in Life Science Leader magazine. But to whet your appetite until its publication in December, you can digest some of the thoughts, predictions, and desires for the space from Omega Therapeutics, Eli Lilly, Arrowhead Pharmaceuticals, and Strand Therapeutics executives in my previously published 2023 Outlook article.
  • If you require further sustenance, look no further than this hot-off-the-press article in which the authors compare Moderna and Pfizer’s respective vaccines, investigating how their ionizable lipids, untranslated regions, and nucleotide modifications impacted each vaccine’s delivery, protein expression, antibody generation, and stability. 

Beep Beep! ~The Road Runner

  • Personally, I always rooted for the coyote.
  • If you’re as peeved about the quality of your AAV as I am the survival of The Road Runner, be sure to join me, REGENXBIO’s Craig Malzahn, and Regeneron’s Andrew Tustian on November 7 at 12 PM ET as we discuss the roadmap to higher quality AAV. You can register to join us here!

Have I come this far, only to strike a wall of stone? ~ The Silver Surfer

  • If you listen closely, you can hear every autologous cell therapy company cracking its knuckles.
  • There’s been a lot of debate about the merits of decentralized or point-of-care cell therapy manufacturing in knocking down the “walls of stone” barring more efficient patient access. I appreciated this LinkedIn article (and its comments!) laying out the biggest questions we have yet to answer and in what ways we can be proactive to make further advancements toward a decentralized model “prototype.”
    • mRNA folks, don’t check out just yet! Though cell therapies and mRNA therapies may be vastly different in terms of manufacturing, personalized mRNA medicines (i.e., cancer vaccines) will still face many of the same logistics and operational challenges as cell therapies.