From The Editor | January 12, 2024

ARW's ATMP Manufacturing Must-Reads (2024 NEW YEAR'S RESOLUTIONS EDITION!)

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

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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. Once a month, 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.

As this was the first must-reads of 2024, it only seemed fitting that we embrace the “new year, new goals” mentality. Because one of my “resolutions” was to be more generous in the new year, I’ve taken the liberty of grouping your must-reads together in accordance with some resolutions I’ve drafted just for you! I know, I know — I shouldn’t have. And you’re very welcome.  

Resolution #1: I will be a champ and subscribe to ARW’s new publication, Advancing RNA.

Resolution #2: I will finally get in shape and run a 4-minute mile!
[RNA 2024 OUTLOOK]

  • And I, ARW, will be the first to send you flowers when you’re admitted to the hospital.
  • Even better, reading my latest Q&A on how to break RNA’s 4-minute mile featuring CEOs and CSOs from Ethris, Replicate, Orbital, and Combined Therapeutics will most likely not require you to add any physical therapists to your speed dial in the new year.
  • My Q&A above may be a quick sprint, but if you’re looking for more of a marathon read, this long-form article in Nature by several of our friends at Pfizer does a very thorough job exploring the past, present, and future of coding and non-coding RNA therapeutics. Stay hydrated.

Resolution #3: I will not expect people to immediately recognize and celebrate the wonder that is me.
[ATMP 2024 OUTLOOK]

  • But if they don’t, it was “nice” to know them.
  • The holidays may be over, but it’s still the season to look beyond ourselves and to take stock of the predictions and expectations for the broader ATMP space in 2024, starting with:

Resolution #4: I will pay off my debt and start saving more money this year.
[MONEY]

  • Thank Christ for scratch-off tickets.
  • ... And also for this new private life sciences growth fund. Goldman Sachs announced its entrance into life sciences investing with the close of West Street Life Sciences. The fund’s initial fundraising efforts brought in $650 million and will be doled out on early- to mid-stage therapeutics, tool providers, and diagnostics companies, specializing in precision and genetic medicines, cell therapies, immunotherapies, synthetic biology, and AI.

Resolution #5: I will educate all my family and friends about the importance of global access to ATMPs.
[MARKET ACCESS]

  • But in case that doesn’t work, any chance you have a connection to Oprah?
  • IQVIA came up with “7 key indicators of progress in the life sciences sector” for its blog. #1 is worth noting, but I’d particularly direct your attention to #3, which homes in on the recent sickle cell gene therapy approvals. In particular, Murray Aitkin argues that a key progress marker for the life sciences space in 2024 will be reaching more than 2% of the sickle cell patient population (i.e., 2,000+ patients) with the newly approved sickle cell disease CGTs.
  • Continuing along those lines, I thought this MIT Technology Review article was a great companion piece to the IQVIA article above — this time presenting a patient’s perspective of what it was like to participate in Vertex/CRISPR’s exa-cel clinical trial. Though the experience/therapy changed his life for the better, the process wasn’t all sunshine and roses, either. There’s a lot to chew on here.

Resolution #6: I will finally accept that self-regulation is just not a thing I was put on this earth to do.
[REGULATORY]

Resolution #7: I will FINALLY start that sci-fi novel I’ve been planning since I was 14.
[mRNA ‘-ICITIES’ & ‘-ILITIES’]

Resolution #8: I will be less litigious in 2024.
[LEGAL ISSUES]

  • Unless your name rhymes with “Karepta”or “Bregenex.”
  • In which case, buckle up: A district court in Delaware has invalidated Regenxbio’s/Upenn’s '617 Patent' protecting host cells containing certain AAV sequences. Sarepta, who was accused of infringing this patent with its AAV DMD gene therapy, has emerged triumphant. (An appeal is most likely forthcoming.)