Taking an Industry Pulse at Meeting on the Mesa

In just two weeks, many in the gene and cell therapy field (including me) will be heading to the Alliance for Regenerative Medicine’s 2024 Meeting on the Mesa in Phoenix. I’m looking forward to connecting with peers and enjoying the warmth of Arizona in October. More importantly, though, the annual event provides an opportunity for me to take a pulse on the advanced therapies industry.

I’ll be paying close attention to sessions with key United States Food and Drug Administration (FDA) experts Scott Gottlieb, M.D. and Nicole Verdun, M.D., as well as Yoshiaki Maruyama, Ph.D. of Japan’s Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA). Regulators have made it clear that they aim to address key challenges to the development of gene and cell therapies, especially for rare diseases, and they want to work with developers in this effort. The Advanced Medicine Partners team has seen firsthand just how critical a robust CMC package is for getting new gene and cell therapies on the market and in the hands of patients. All too often, developers get slowed or even halted by immature manufacturing processes that produce drug product with unacceptable levels of process and product-related impurities. Choosing a CDMO with experience can ensure a drug substance of optimal quality is produced. I’m curious to hear what Drs. Gottlieb, Verdun and Maruyama will add on this topic. Additionally, I believe we’ll hear from them that increasing alignment between FDA and ex-US agencies, like PDMA, should be prioritized to increase the efficiency with which advanced therapies are approved.

Outside of regulatory discussions, I’m eager to spend time interacting with developers during my time in Phoenix. Learning about novel capsid programs being pursued informs how I (along with the full Advanced Medicine Partners team) can best support potential partners. We take pride in our approach of using the proven “spine” of our manufacturing process along with bespoke elements tailored exactly to the needs of a specific program. Personally, finding unique and creative manufacturing solutions without ever compromising on quality is one of the best parts of my job. I also like focusing on helping newer AAV-based companies advance their programs rapidly and cost-effectively, again maintaining the highest quality. Advanced Medicine Partners has developed a robust and reproducible manufacturing process that is commercially viable and highly productive for AAVs, including best-in-class analytics and characterization. Our innovative AAV process is one of the most productive in the industry and consistently generates >95% functional full capsids.

If you’ll be in Phoenix, stop by the Advanced Medicine Partners booth (FLW Building, Table F21) and join us for our CEO Andrew Knudten’s presentation on Tuesday, October 8 at 10:45 a.m. MST in FLW Ballroom G. I hope to meet you in person in just a few weeks, but if you aren’t going to be at the Meeting on the Mesa, you can reach us at partnership@ampgtx.com.

Joe Balleydier
Senior Vice President, R&D and Innovation