By Anna Rose Welch, Director, Cell & Gene Collaborative
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I recently came across a KPMG report entitled “‘One-and-done’” Gene Therapies: A Near-Term Niche?” The report’s focus was more commercial/strategic in nature, with only a brief high-level look at manufacturing issues — most of which will hardly be news to those of you on the manufacturing floor. However, the authors’ overall takeaway left me thinking about what this could mean for manufacturing professionals.
As the authors explain throughout the report, it’s the Wild West out there for modalities and technologies, and it’s going to be a waiting game to see which will emerge as the winners (yes, plural). As such, they urge companies to stay the course, spread their bets across the technologies (if possible), and focus on establishing a long-term presence in gene therapy.
Staying the course could mean a lot of things, including eating ice cream at midnight to brace for the dawn (#NoJudgement). Disillusionment inevitably goes hand-in-hand with excitement in any nascent industry. (Just ask me about my time in the biosimilars market.) Between urgent patient need and the prospect of intense competition, there’s a fire under each advanced therapy company to be first to the market. But there’s also something to be said about being part of the second wave (a “fast-follower”) to the market, especially when we think about it from a COGs and technological innovation standpoint.
No one has — or really could — explain this better than Deanna M. Peterson, CBO of AVROBIO, who appeared on a recent episode of the Ori Spotlight Podcast. As she noted, all eyes are locked onto the late-stage companies because they’re “leading the world into the commercialization of the first gene therapies…. We’ve all been taught that being number one in our industry is good, but sometimes being number two is pretty good because you can sit back, watch [the leaders] bear the brunt, make the mistakes, test different ways of doing things, and then advance the field….”
Even as recently as the past few years, a variety of solutions have crept onto the market enabling companies to stay lean infrastructure-wise and better manage their financial and technological resources. Look no farther than logistics companies that assist in delivering ex vivo therapies to patients or emerging fully automated platforms enabling a plug-and-play approach. With time, greater sophistication will also be applied to modeling COGs across adherent and/or suspension-based cell culture technologies for vector development (as this goldmine of a paper begins to explore).
These days, CMC has to hit the ground running even before a C&G company is launched. But there’s something to be said about being earlier in your commercialization journey as the market is on the cusp of welcoming its first wave of gene therapies. You may not be at the market finish line right now (or next year) but this affords you (and tech providers) the time to fine-tune, de-risk, and implement new strategies and tools that will benefit your overall COGs in the long run. And if there’s one thing we do know, better control of COGs in and of itself will position you as market leader in this space.