Policy Summit

Your Guide to a Can’t-Miss Policy Summit Session on CMC and Regulatory Considerations

Emily Walsh Martin, Ph.D. - August 21, 2020

Core to the success of any gene or cell therapy development program is a well-thought-out plan for manufacturing which bridges everything from early discovery selection of a candidate construct to your process validation runs to support a commercial application. 

Don’t read any further.

Seriously, don’t. Go to your calendar and make sure you have blocked 3 p.m. Central Time on September 23. I’ll wait for you to return.

OK. We’ve got that out of the way. I’m glad, because I would hate for this session to be over-scheduled by a Zoom meeting at work.

Here’s the thing, and I think we all appreciate this, but sometimes need reminders. Core to the success of any gene or cell therapy development program is a well-thought-out plan for manufacturing which bridges everything from early discovery selection of a candidate construct to your process validation runs to support a commercial application. 

The complexities of that statement can’t be underestimated. 

It is an enormous challenge to understand the full path when you are only in the earliest stages of designing your therapy. You have so many unanswered questions at that point. What is my manufacturing platform today? Will it be the same later? Who will manufacture for my IND-enabling studies? Clinical studies? Commercial product? How will I establish comparability between those products if I change manufacturers? What are the key attributes of my drug product that impact its safety and tolerability? How do I ensure that they won’t drift over time as I manufacture more batches?

And there are always pressures along the way. Funding limitations, raw material limitations (e.g., patient PBMC mobilization variability), timeline considerations, manufacturing slots, GMP documentation requirements, retain requirements, stability programs, device compatibility programs, failed batches, batches that don’t match yield expectations, material requirement changes based on regulatory feedback, volume of administration limitations, concentration limitations, to name just a few. And we know this means on-the-spot decision making about what to prioritize now, and what that might mean for the program later.

So, if you have been sitting there in your pajama bottoms thinking “Gosh, I wish I had a better view of the big picture of what really matters long term” (i.e., beyond a successful IND filing), then the CMC and Regulatory Issues session of the ASGCT Policy Summit is more than worth the price of admission.

Now maybe you are thinking: how are you so sure, Emily?

Well, I got a preview of the presentations that Dr. Herb Runnels (Pfizer), Dr. Katie Miller (Dark Horse Consulting), and Dr. Denise Gavin (OTAT CBER FDA) have planned for us. And not only do they plan to highlight their deep knowledge about key considerations around critical quality attributes (CQAs) of gene (Herb) and cell (Katie) therapy, but also they will speak to the global convergence of thinking around CQAs from an FDA perspective (Denise). Moreover, with the expert steering of the session chair, Dr. Jan Thirkettle (Freeline), 50 percent of the time will be devoted to audience questions (i.e., your questions!)

Where else can you find a panel willing to discuss not only specifics around CQAs but also a framework for defining them? Where else could you hear experts ponder the paradox of the challenges of establishing comparability between batches without understanding CQAs all the while that the biology is still being worked out in the studies for which you need to establish comparability?! 

Nowhere else. So register now. And do not miss this most informative session. I will see you there!

Dr. Walsh Martin is a principal at Tremont Therapeutics and a member of the ASGCT Communications Committee.

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