Takeaways from Cencora’s inaugural Cell and Gene Therapy Summit

Cencora: The summit had very rich and broad sessions. What did you find most surprising or noteworthy about the discussions? 

Louis: I’ll start with a positive observation, which is the admirable level of collaboration between developers in the industry. It was clear they know each other, meet regularly and communicate. Even those who are technically competitors have an attitude of being in it together. On a more critical note, the patient journey remains surprisingly riddled with communication gaps. There is a lot of information available in the field, but the challenge is gathering it, interpreting it, disseminating it in a way that's digestible to the patient and it’s clear from patient surveys that there’s a lot of work to do.  

Lung-I: What was particularly significant was the recurring theme of market access, which was not only discussed in the sessions dedicated to this issue, but also in the commercial panel that I moderated, in the regulatory panel and other sessions. This underscores the importance of patient access, a topic that is top of mind for many, and the various challenges it presents, including geographic limitations and the complex pricing and reimbursement discussions that are crucial for all stakeholders.  

 

Cencora: There has clearly been a lot of progress in cell and gene therapy development, which came across in the summit. What stood out for you as the most optimistic developments? 

Lung-I: Perhaps most notable is what Dr. Nicole Verdun (Super Office Director, CBER, FDA) talked about regarding guidance around platform technologies. This was also a hot topic at the BIO Convention (held in San Diego from 3-6 June). It is exciting because it could accelerate the development of CGTs in the U.S. It would allow manufacturers to leverage data from approved therapies to iterate and improve the technology faster. I was encouraged to see this progressing so quickly, as it had just emerged as a topic on the pharma side a few years ago.   

Louis: The field is maturing fast, with a lot of knowledge and data available across the board. There are discussions about study design and how to optimize that, long-term follow-up strategies, and real-world evidence. There are also some successful patient services solutions being discussed and celebrated.  With so many resources at our fingertips, the focus now is figuring out how to make the best use of the information to build a strong value story, improve the patient journey, and optimize drug development. 

 

Cencora: Let’s go back to the session with Dr. Verdun. What is your takeaway in terms of what the regulators are doing to advance development? 

Lung-I: Aside from the platform technologies, the FDA is open to talking to and helping manufacturers.  Manufacturers are sometimes hesitant because they are concerned about having the perfect data and having their program sorted before meeting with the agency. And yes, you want to go to the agency with your ducks in a row, but it’s noteworthy that the agency is very collaborative and willing to work with the industry.  

Louis: I noticed that companies in various stages of development have differing views on the evolution of requirements, particularly regarding elements like CMC and manufacturing considerations. That’s why it’s important to think about these programs holistically and start with the end in mind, but understand what is required at each stage. When it comes to the regulators, there’s a clear willingness to talk to manufacturers early on and help to eliminate some of the unknowns and to close the communication gaps.  

 

Cencora: What stood out to you as being a big concern for the industry? 

Lung-I: There are still concerns around funding, especially for smaller companies that have had to make tough decisions. Hopefully, we’ve hit bottom, and it will get better from here, but I think funding is top of mind, at least for now. For bigger companies, there’s a need to think about how they will grow. Some gene therapy companies are doing quite well, while others have plateaued. For those big companies with products on the market, the focus needs to be on how to grow and, in some cases, compete.  

 

Cencora: What do you believe the learnings from the summit mean for stakeholders generally and where cell and gene therapies are headed? 

Lung-I: To me, it's communication, collaboration, and competition – the three Cs. You need to have communication to learn and propel the field. We talked about collaboration and how that is happening in the industry. And then competition – by which I mean friendly competition. To enhance patient access, you need to have more therapies available. There are at most two therapies for any given indication, but we can do a lot more. So, we need to focus on those areas. One other significant development is the use of technology to streamline the manufacturing process and take more manual steps out of this process to bring down costs and speed everything up. And competition will induce those technology developers to develop more innovations to advance production.  

Louis: We’re seeing new ways to manufacture and new technologies blurring the line with cell and gene therapies. One thing that stood out for me was Lung-I’s fireside chat with Rita Johnson-Greene from Alliance for Regenerative Medicine, where they talked about different types of technologies in various disease areas. So, there’s a lot of exciting stuff on the horizon.  

 

Cencora: How would you sum up the overall experience from the first Cell and Gene Therapy Summit? 

Lung-I: Seeing the engagement and willingness of so many to speak and spread knowledge was great. And this is unique in the industry. Cell and gene is a community, and those of us in it are all looking to do what we can for that community, which is great to see. 

Louis: Building off that community theme, what struck me was the huge diversity in terms of fields of expertise. We covered many different areas of drug development, commercialization, regulatory, patient engagement and access. Our audience heard discussions and opinions they wouldn’t necessarily have been exposed to elsewhere. And that’s important in a field like cell and gene therapy where you need to think holistically about your program for the best chance of success. For me, that was one of the most powerful takeaways from the summit.  

 

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