By Anna Rose Welch, Director, Cell & Gene Collaborative
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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.
Lucky for us, this newsletter was sent on July 13, which is smack dab in the middle of Sports Cliché Week (July 11-17). As such, I stepped up to the plate and carefully selected a sports cliché to accompany your manufacturing must-reads. Here’s hoping you think at least one of these selections hits it out of the park. Play ball!
You gotta want it!
[PDA ATMPs RECAP]
The ball’s in your court.
They’re in a league of their own —
- And so is every person’s definition of “flexibility” when it comes to manufacturing technology.
- Hence why I took the chance to pick the brains of two executives — one from a cell therapy biotech and one from a tech provider — about how they are defining flexibility in cell therapy manufacturing platform technology and what this may mean for technology partnerships in the broader CGT space.
We must play within ourselves.
- Is it just me, or can we turn this cliché into an mRNA joke?
- As I am no doubt the only one that finds this “joke” clever, allow me to offer you something infinitely smarter — a recent paper from Advanced Drug Delivery Reviews walking us through the current understanding of mRNA’s structure, the IVT reaction, RNA polymerases, capping agents/strategies, and future RNA technologies (i.e., saRNA and circRNA).
- This week, partners Merck & Moderna just announced their plans for an 1,100-person pivotal trial for their cancer vaccine in combination with Keytruda. As such, it seemed like a great opportunity to share this publication from Stanford scientists on some of the latest developments in the mRNA cancer immunotherapy space.
- In addition to Merck and Moderna’s big news, I’d be remiss if I didn’t share the news out of Cartesian Therapeutics published last month in The Lancet: The biotech’s autologous RNA CAR-T therapy showed its skills playing offense against Myasthenia Gravis in a Ph.1/2 trial, making it the first (so far successful!) study of an rCAR-T in an autoimmune disease indication.
He brought his A-game today.
[SHAMELESS COWORKER PROMOTION]
- The first official interview and first published piece of editorial is a big rite of passage here at Life Science Connect.
- Today, I’m celebrating my newest editorial colleague Tyler Menichiello, not only because he recently published his first Cell & Gene article on some of the challenges of licensing ATMP platforms from universities, but also because he didn’t pull any punches and carried out that interview in-person while visiting a Pittsburgh biotech.
On paper, they’re the team to beat.
[AAV MARKET RESEARCH]
- In the ATMP world, it’s not just about looking good on paper — it’s also about how a product is formulated and how long it can last on a shelf at room temp.
- Special thanks go out to one of my favorite Cell & Gene Collaborative “geneiuses” for tipping me off to this trusty “AAV Formulation Cheat Sheet,” outlining the stability, concentration, and excipient profiles of currently approved viral vector therapies.
They have all the right pieces.
It’s a slam dunk!
- Someday, hopefully, we’ll be able to say this about the delivery of our ATMPs — particularly using nonviral delivery mechanisms.
- This article in Nature is a nice review of how several different gene therapy biotechs are playing with nonviral delivery methods, whether they be LNPs, DNA or protein-based nanoparticles, exosomes, or an array of physiochemical tools (for ex vivo products).
- Speaking of exosomes and peptide-guided delivery — catch!