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.
Valentine’s Day may still be a few weeks away, but this newsletter was sent out on National Spouses Day, which gives us another valuable opportunity to lavish our significant other (or cat) with praise and gratitude. So, in addition to your must-reads below, please also find several “compliments” that will, no doubt, be just what your partner wanted/needed to hear today.*
*Please note, these “compliments” are simply recommendations. A risk-based approach is advised.
[2023 ADVANCED THERAPIES WEEK TAKEAWAYS]
“Whose heart has four chambers and keeps beating when you’re around?”
- For the full effect, make sure you remember to raise your hand when you say this to your person/cat.
- After returning from a whirlwind week in Miami at Phacilitate’s Advanced Therapies Week, I put the strength of my own heart to the test by hitting the ground sprinting and penning this article outlining my four biggest takeaways from the conference. Given that one of these takeaways actually sparked a (friendly) bioprocess-related debate on LinkedIn, I have affectionately labeled them “Hot Takes.”
- Whether you find them “hot” or not, however, well... feel free to keep that to yourself.
“I love it — so much — when you
talk endlessly tell me everything about your job.”
- I’m so glad that’s something you like, because I just wrote an article about one of the biggest challenges the mRNA therapeutics space is facing — and it has a lot to do with what I do every day. [Metaphorical mRNA: An Argument For (More) Creativity In RNA Therapeutics]
- But this isn’t just ANY article; it also serves as the official introduction to a new passion project of mine — a creative video series, ARW on RNA — which I’ll be launching next week.
- If reading (anything more than this newsletter) is going to exhaust you to the point you won’t be able to pay homage to your spouse and/or pet turtle later, please — for their sake and yours — cut straight to the chase and watch the 60-second “teaser” video for ARW on RNA.
“You look really nice today. What did you do differently?”
- While you (frantically) attempt to determine your partner’s current “MOA,” take comfort in the fact that you’re at least in good company. The entire ATMP space is just as bewildered as you are about the thing(s) their products did/are doing — and why.
- If you still can’t immediately tease out whether your partner is rocking a new haircut or a shockingly brilliant new set of teeth, you may be interested in learning some tips on characterization from this whitepaper by the good folks at Dark Horse Consulting. Here, they pull back the curtain on rAAV product attributes and product-related impurities, as well as share analytical characterization approaches to better understand and demonstrate rAAV heterogeneity.
- Following an ARM/ASGCT/FDA working group session on potency assays, I put together an article boiling down the industry’s and regulators’ most prominent questions, concerns, and opinions on clearly and efficiently demonstrating potency. Hint: It’s a bit of a nightmare for everyone. [CGT Potency Assays: When Will We Wake Up From The Nightmare?]
- The discussion on potency/analytical development continues in this executive summary from USP’s Biologics Stakeholders Forum 2022 on CMC challenges for CAR-T therapies. Though it’s just a brief high-level summary, it should be reassuring that your colleagues across the industry are just as vexed as you are by raw material and product characterization and assay validation. There are also a few suggestions on how to go about tackling that bedeviling matrixed potency assay.
“You never cease to amaze me — I can tell exactly how much I screwed up just by looking at your face.”
- While unspoken communication in relationships is transformative (for better or worse), we unfortunately can’t get a product approved simply because our CMC reviewer told us something deeply meaningful with their eyes yesterday.
- My colleague Erin Harris at Cell & Gene just hosted a live event featuring two fantastic regulatory affairs experts from REGENEXBIO and BridgeBio on some of the biggest regulatory impediments facing the CGT space today.
- There are some additional, valuable regulatory-centric tidbits shared throughout this article by ASGCT, summarizing presentations from the FDA OTAT and ASGCT annual liaison meeting late last year. In particular, I’d call attention to the section within which the FDA’s Wilson Bryan lists some of the most common reasons why RMAT designations are so hard to come by today.
- There’s also a section on ASGCT’s thoughts on where we could stand to see some greater regulatory clarity when using novel engineered AAV capsids.
- In case you missed it, PIC/S released its 2023-2027 strategic planning document a few short weeks ago. You can check out a really quick bulleted summary of what the document covers (as well as the document itself) in this LinkedIn post from Daniella Drago who, by the way, is a must-follow if you like staying up-to-date on the world of regulatory affairs.
“Is that you I smell?”
“You’re worth way more to me than silver and gold. By the way, how’s that raise coming?”
- I mean, to be fair, economic stability is supremely sexy.
- On the biopharma funding front, my colleague Ben Comer of Life Science Leader penned an outlook article asking life sciences CEO’s their take on what the year ahead will hold for alternative funding models, M&A trends, financial risk management in R&D, and more.
- For a more specific look at the CGT and mRNA spaces, the cool kids at DeciBio recently released a modality-specific infographic showcasing data from a survey on anticipated growth in a variety of key areas (e.g., headcount, clinical trial initiation, M&A, new funding).
- Interestingly, despite concerns about funding overall, respondents expressed optimism that oncological-focused cell therapy companies, CNS-focused gene therapy companies, and a variety of indication-tackling mRNA/RNA therapeutics companies are likely to receive funding in the year ahead.