ARW's C&G Manufacturing Must-Reads

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 C&G therapy manufacturing, in particular, and/or other C&G 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. But inboxes can be shifty places. (Cue George Carlin on “Losing Things.”)

So, here’s a “permanent” copy of my C&G Manufacturing Must-Reads newsletter that was delivered on August 26th, for all you practical people who, like Carlin, abhor the question, “Where is it?”

The Picasso Effect: The Many “Angles” Of The C&G Regulatory Pathway

• A simple question to the FDA’s Peter Marks at the WMIF about how the agency establishes its cell and gene therapy-specific guidance sent me on a winding (but enjoyable) journey into where the regulatory pathway currently is, where it needs to be, and how the industry is striving to arrive at a more streamlined CMC package. Check out part 3 of my ongoing regulatory series on the Cell & Gene Collaborative blog.
• If you’re looking for a good high-level look at the industry, ARM just published its Regenerative Medicines In 2021 report, which covers all of the funding, regulatory approval, clinical/scientific, and policy milestones you’ll ever want to know. It’s “manufacturing-lite;” however, ARM is actively involved in shaping Cures 2.0, sections 303 and 308 of which call for, 1.) an HHS report on the state of the C&G industry and foreseeable future FDA regulatory challenges/strategies (page 59-60); and 2.) FDA guidance on CMC development and review standards for breakthrough/RMAT-designated programs (page 69-70).
• The FDA and rare disease makers are holding strong as criticism against the accelerated review pathway revives following the aducanumab decision. FYI, back in 2019, Scott Gottlieb pointed to the accelerated review pathway as a “promising” opportunity for gene therapies, in particular.

If You Give A Mouse Patient A Cookie Gene Therapy: The Cause & Effect Of No Patient Access

• This editorial published in Nature Medicine entitled “Gene Therapies Should Be For All” came out at the perfect time, following the news that Bluebird Bio and its gene therapy Zynteglo will be retreating from the EU market. Though this is disappointing on a lot of levels, I had some thoughts on why this is especially sad news on the manufacturing front.
• After attending a recent industry panel, I penned an article singling out two of the biggest lessons the ATMP industry has learned from COVID-19. In particular, I share some thoughts on the role you manufacturing masterminds have in helping the industry get the Aretha Franklin-level R-E-S-P-E-C-T (and patient access) it so deserves.

“The Delivery Games:” Put Katniss Everdeen & FedEx Overnight Shipping To Shame 

• A few months ago, McKinsey released a report on the current and future landscape of viral vectors. Little did McKinsey know, my biggest takeaways for industry success were around how to keep your vectors from being little brats.
• Lenti peeps, I told you last time I’d deliver the goods (pun fully intended). If you spend your days wondering which cell culture technologies are the best for lowering your COGS/dose, I have found you the holy grail of publications. Better yet, not only did I summarize said publication for a quicker read, but I am delighted to report that the accompanying sonnet basically wrote itself. FYI, AAV folks, the cost analysis tool described here can be extrapolated to AAV production.
• Tired of viral vectors and lipid nanoparticles getting all the love? A new publication in Science (also reported here in STAT+) showcases some very early stage but still intriguing findings on a (possibly less immunogenic) means of delivering gene therapy/gene editing technology to different cells and tissues. Let’s press SEND.

Follow The Leader: Words Of Wisdom From Your Counterparts 

• Counterpart is not just an incredible show on Amazon Prime (seriously, check it out), but your actual C&G manufacturing counterparts are pretty fabulous themselves. In these two Q&As, Danielle Appelhans (former CTO of Novartis Gene Therapies) and Biogen’s Miles Devany, site head and general manager of gene therapy manufacturing, share their best leadership lessons.

Ancillary Materials 

• Eerily long necks, bone-carved apple scoops, and (semi-) illicit mango sourcing are just some of the treasures I’ve found for you in the past few weeks. Check out my brief round-up of (non-cell) culture-related stories here.