From The Editor | October 19, 2023

ARM's 2023 Meeting On The Mesa: Data, Ethics, And More


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

cell gene meeting on the mesa

Last week, I attended ARM’s 2023 Meeting on the Mesa in Carlsbad, CA. More than 2,000 cell and gene therapy leaders were in attendance, and as usual, the event was chock full of informative sessions, impactful conversations, partnering opportunities, and networking, networking, and more networking. In his keynote address, ARM’s CEO Tim Hunt shared that the event has outgrown the current venue – a topic I had the pleasure of discussing with ARM’s COO, Rita Johnson-Greene. Rita and I recorded an episode for Cell & Gene: The Podcast on site at the event; among many other Mesa-related topics, Rita shared some teasers about next year’s event. Subscribe to Cell & Gene: The Podcast now so you don’t miss our conversation.

Can We Afford Gene Therapy?

This is my third Mesa in person, and I always leave the event far more knowledgeable on the current state of the CGT landscape than when I arrived. Some of the high notes from the educational sessions included Tim Hunt’s keynote address, which shared telling stats about the state of the CGT space. For example, in 2023, we may see 8 approvals and potentially 10-20 approvals by 2025.

Cost has always been a major concern in CGT. That said, Hunt assured the audience that, YES!, we can afford gene therapy. Data suggests that gene therapies/gene editing medicines are projected to cost the U.S. healthcare system $7.5 billion in 2030 (~ 18B globally). To put this figure into perspective, Hunt shared additional data for context. This is one-tenth of one percent of projected U.S. healthcare spending in 2030. Also, 1.3% of projected U.S. prescription drug spending in 2030; 1% of projected federal spending on Medicaid in 2030; 1.8% of medical costs for rare disease patients in the U.S.; and comparable to the amount of Medicare spent on one diabetes drug in the last year. In short, healthcare systems should be able to digest the coming wave of genetic medicines. Promising news for CGT companies and patients.

The Importance of Ethics in Cell & Gene Therapy

Of the many valuable sessions during this year’s Mesa, I found the session on ethics to be one of the most interesting. Tim Hunt moderated, The Importance of Ethics in Cell & Gene Therapy, featuring Arizona State University’s Benjamin Hurlbut, Ph.D; Former ARM CEO, Janet Lambert; Life Science Cares’ Rob Perez; Novartis Gene Therapies’ Tay Salimullah; and Canadian Organization for Rare Disorders’ Durhane Wong-Rieger, Ph.D. During their time on the main stage, the panelists covered consent, cost structure, etc. This was one of those sessions where solutions weren’t reached, but the conversation itself is important and long overdue.

Regarding the overall importance of ethics in the CGT field, Rob Perez stated, “Ethics is defined by a set of moral principles that helps you to navigate very difficult and challenging problems and situations. As a former CEO, and as a founder, there are many, many times where having an ethical compass really does help to make the complex simple. And I think that’s the way I would think about ethics for this panel and ethics for this industry; we are dealing with extraordinarily complex, extraordinarily challenging issues. And if we can have more conversations and come to more alignment on what our ethical standards could be for the industry, it can help to be kind of a North Star on how we want to operate and how we want to make those very difficult decisions. And for me, that’s always been an important part of how I can deal with the most challenging questions, the most challenging decisions, and the most challenging complexities in an operating business.”

Durhane Wong-Rieger, Ph.D. shared, “For me, it’s always helping people to understand what the scenario is for themselves, and day in and day out, to get to the best decision for each person. So, we can’t always do what is ethical, but we can always do what is good. In fact, that whole notion of “do no harm” is sometimes our best ethical principle. When asked is there an ethical solution? I would have to say, I have no friggin’ idea. I absolutely don’t know. And I think that’s because we really don’t have rules for this in terms truly lifesaving therapies. The problem is we can’t give them (i.e. lifesaving therapies) to everybody, or we even want to give them to everyone, because not everyone wants them. So that’s also an issue here. Give somebody a lifesaving therapy if they don’t really want to take it, and you fault them because they don’t want to take it.”

Partnerships, Data, Cost Savings Top of Mind for Biotechs / CDMO Partnerships

I had the opportunity to talk to quite a few biotech leaders about their interest in partnering with a CDMO. Unanimously, our discussions centered around data, cost-efficiency, specialized expertise, and risk mitigation as primary reasons for partnership. The heaviest emphasis was placed on cost savings and data. Cash is tight right now, and biotech leaders realize that partnering with a CDMO with the specialized expertise, as well as infrastructure, cost-efficiency, and risk mitigation know-how can help CGT companies bring their therapies to market successfully.

The biotech leaders I spoke to were loud about the fact that data is critical for partnership with a CDMO. Biotech leaders also want a partner that collects and analyzes all data. Process optimization data; data to help biotech companies refine and improve their manufacturing processes to achieve higher yields, better product quality, and cost-efficiency; data that are essential for regulatory submissions; data on scaling up production processes; technology and innovation data. If the conversations I had are any indication of biotech’s manufacturing needs, secure and accurate data is a must for partnership with a CDMO.

I’m already looking forward to the 2024 Meeting on the Mesa scheduled for October 7-9 in Phoenix, AZ. Until then, be sure to subscribe to Cell & Gene: The Podcast wherever you get your podcasts, as I recorded episodes at this year’s event with Astellas’ Richard Wilson and Aurion Biotech’s Greg Kunst, and of course, ARM’s Rita Johnson-Greene. A new episode drops every other Thursday.

Did you attend this year’s Mesa? Let me know your takeaways from 2023 Meeting on the Mesa and how you’ll incorporate your learnings into your role.