Moving Cell and Gene Therapy Towards the Future: Q&A with Maher Masoud

Cell and gene therapies (CGT) continue to advance in the pharmaceutical industry. Despite continued investment in R&D, the space still faces significant barriers. Primarily, the expensive nature of the treatments and complexity of manufacturing them.

Maher Masoud, CEO of MaxCyte, spoke with Pharmaceutical Executive about the CGT space and how the industry is working to move past these barriers. Advancements in platform technology are allowing companies to more efficiently partner with manufacturers to improve those processes, while new areas of research (such as non-viral cell engineering) are opening new possibilities.

Pharmaceutical Executive: What will it take to scale cell and gene therapies from discovery to commercialization?
Maher Masoud: What it takes in this environment is developers that can work with manufacturers that have the ability to scale from R&D to commercial. What I mean by that is working where you're not having to re-optimize processes on a regular basis and not having to start from scratch every time you're working with a manufacturer.

That's what it’s going to take for this industry to have developers come together with best-in-class platforms that have the ability to do optimized processes early in research, and then translate that from that early research optimization into a clinical platform so that a clinical process that can make it all the way to commercialization.

In this environment, you must work with across-the-board, unit manufacturing operations, where you're recreating the wheel each time. It makes it very difficult for developers. The science is there.

If you look where we are right now, we have a lot of curative products out there for oncology and rare genetic diseases. But being able to work with developers that have done it in the past, and have been in the clinic multiple times, is going to be the key for what developers need to do, which is work with seamless, automated and scaled up processes.

PE: How is the role of non-viral cell engineering technologies growing in advanced in therapies?
Masoud: It's going to continue to grow. What we're seeing in advanced therapy is the modalities and the number of complex aiding required is only increasing. Right as we go from your basic one edit CAR Ts of years past, you're seeing modalities now where we're doing multiple edits, knock-ins, knockouts.

With cell therapies, that's where you're doing everything outside the body. And on top of that, the reason why it's so critical is from the quality analytics and quality control perspective. With cell therapies and non-viral cell therapies, you can control the safety of the final therapeutic product, and that really lends itself to cell therapies.

It's only going to grow from here. We had some headwinds in years past, but we feel very confident cell therapy, especially non-viral, is where medicines are heading.

PE: How are platform technologies and partnerships shaping the CGT development ecosystem?
Masoud: It's coming together. Platform technologies are shaping it and we're working towards having many different technology providers and platforms come together and work together.

With CDMOs, we can streamline the process and the entire manufacturing process for developers and hopefully get to a day (especially for autologous therapies) where you're dosing patients on a patient-by-patient basis.

You can have more manufacturing done closer to the patient. So, by having developers come together and having manufacturing operation providers and enabling technologies come together to work with CDMOs closer to hospitals, we can make patient accessibility much better than it is right now.

PE: What lessons have you learned in the current biotech funding environment?
Masoud: Biotech funding can be a little bit lumpy at times. That's not new. We've seen this in the past. You have your tailwinds, and headwinds at times. What we have learned (and it's actually a good thing) is that the headwinds we've seen the past few years from the biotech industry have refocused the industry.

A lot of developers and companies are focusing on priorities. What makes you really great at what you do? Focus on what you're great at. And then for developers, what we're noticing is the funding environment, even though it has had a headwind, it's allowed them to focus on lead assets.

Rather than trying to take multiple programs into the clinic and have to continue to be on the cycle of trying to raise additional funds, focus on the science. See what your lead asset is and what you believe you can get through the clinic and to commercialization. In essence, they've sharpened their pencils in this environment, and we're seeing that.

What we're also seeing (and I think it's a good thing that we're seeing this) is that the entrepreneurial spirit is coming back. I don't think you need to have as much funding to develop products all the way through the clinic. We've been at conferences and we've heard this from others as well.

Now's the time that we're seeing companies really start to say themselves, do I need to raise as much as I raised in the past? It'll take a product to the clinic. Maybe not, especially if you're focused, especially right now and have the ability to really have come together across industry and share ideas.

We've seen it where you don't need as many funds, you don't need as much cash on hand to take a product all the way through the clinic. I think it's a positive the headwinds we had.