Smoothing the Road to Cell and Gene Therapy Commercialization

To start with, please could you introduce yourself?

My name is Brie Perell and I am a Senior Product Manager for Bio-Techne.

What can early-stage cell and gene therapy developers do to futureproof their programs?

As customers are thinking about transitioning from the research to the GMP scale, they should be thinking about future proofing and really making sure that they’re choosing the right pieces up front that can be transferred into a GMP clinical and commercial scale process.

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So, we offer all our reagents, both at the RUO and the GMP scale, that are manufactured in the same facility, that follow the same manufacturing process to minimize any sort of inconsistencies or differences as customers transition. We also have a quality team and regulatory support team that helps customers as they transition into a GMP product and have the right DMFs to support those customers along that journey.

How can a smooth transition from benchtop to clinic be supported by the right partner?

Our RUO products and our GMP products have that lot-to-lot consistency as customers transition into GMP-grade products, allowing that transition to be a little more simplified and able to scale and transition over. Having the right pieces in place can really help customers move along in that trajectory.

Can closed/sterile cell manufacturing systems advance cell and gene therapy development?

Having the right pieces in terms of closed processing is really going to help customers transition, and so we’ve been focused on developing a lot of our reagents into packaging and processes that are more flexible but also able to be integrated into customers workflows.

How can cell and gene therapy developers plan to scale their manufacturing processes?

As customers are transitioning into GMP processes, we really want to ensure that the process is robust and efficient, but also cost effective, so we manufacture our reagents in a platform to essentially help customers make that path or make that transition as seamless as possible.

So, we partnered with ScaleReady, and with that team that includes our Wilson Wolf team as well as the Frensius Kabi team, we can bring an integrated workflow solution to customers to be able to really enhance the process to make it as simplified as possible.

To start with, please could you introduce yourself?

I’m Josh Ludwig. I’m the Global Director for ScaleReady. That’s our joint venture, which we’ll get into. I’ve been in the cell and gene space for about 11 years and with our core technology, G -Rex, for that time as well. Scientist by training, but it’s been fun helping companies and our clients build their processes and reach commercialization. I’ve been able to start groups early and get over the commercial finish line, and we will hopefully see more and more of those coming up.

How can innovative solutions reduce manufacturing costs?

Reducing costs is definitely the name of the game. We need more treatments to more patients.

We do that by, at least with our technology, especially G -Rex here, our core technology. This is enough cells for one patient and multiple doses for that one patient. We can fit many of these or many patients in a single incubator stack, and so within a given space we can shrink down the amount of space required, or in that same space, ramp up the total number of patients that we get through.

Additionally, because it’s a single-use consumable that doesn’t need to be tied to expensive equipment for that duration, again we’re further dropping the cost down.

As a group, especially here in Miami again, another year gone by, I think we’re all trying to figure out how we lower the cost of these drugs and increase the speed of their delivery.

How can proactive partnerships benefit cell and gene therapy developers?

I think we’ve all seen the need in the space, especially this past year, to be more efficient as a field.

Dozens or a hundred companies trying to solve the same problem 100 times over is very cost-inefficient, especially when there are vendors, partners like ScaleReady, that are creating our technology for them, and we can help them take shortcuts. Basically, we can say, we’ve been there, done that before, so see us more as a partner, ask us questions. Instead of starting from ground zero, we could kind of help them speed along.

A theme that we’re really pushing is if we can help standardize these workflows and standardize the tools that are used, it really speeds everything up and gets access to more patients.

Any advice for early-stage developers working with vendors?

You’ve got to be proactive and got to plan for the clinical success.

If you have not defined your process well and simplified it really well and started thinking about large scale, scale up and scale up production early, these groups are finding themselves needing to go back and generate more clinical data and they don’t have the time or money to do that.

As an industry, again, back to that first question, see your vendors as partners because they’ve been there done that with other clients. They have had similar interactions, they can help you take shortcuts, hopefully, and start thinking about beginning with the end in mind before you end up in a position where you have to backtrack.

Can you speak about ScaleReady’s approach to collaboration?

The tool providers and technology developers that are here in Miami this week, we all have the same goal. We’re all trying to use our special widgets and tools and equipment to allow therapeutic developers to move faster.

If they see us as partners that have knowledge, at least on the manufacturing end, we really know how to manufacture cells. They know how to define the targets and determine what constructs are going to have the best clinical efficacy for the patients, but we know how to manufacture. We’ve been doing this for decades. A strong partnership from a therapeutic developer to vendor is critical, but as we have a lot of vendors here, I think more and more it’s about how we co-develop collateral, co-develop public processes where we can tell people that it’s been done, it’s repeatable. There are tools today that are very effective.

I think as an industry, more and more vendor-to-vendor collaboration, where historically there’s been less of that, I think there’s a bigger appetite now to show the field there are tools out there that are working in commercialized products like the G -Rex, like our instruments here at ScaleReady.

How can flexibility in the manufacturing process benefit cell and gene therapy developers?

It’s the way of the future.

As tool and technology developers, instead of just handing over a tool and saying figure it out, there’s so many options and choices.

It’s like everybody in their day-to-day lives – it’s information overload, so I think we can help curate the technology in a way that makes it easy for them to develop their protocols.

We can show them that you can start on really small-scale instruments and small-scale G -Rex bioreactors, define your process early, and you don’t have to commit to a giant convention center building and equipment until you’ve commercialized.

I think this partnership with Bio-Techne, ScaleReady, and our other partners, Wilson Wolf and Frensius Kabi, we broke new ground when we launched this 3 years ago and we said, as a group, could we come together as more of an altruistic scientific endeavor than maybe where the field was 10 years ago, it was more open sharing of best practices.

I think it’s up to tool providers now to say this internal data that we’re generating, spend the money, do the work in our applications labs, prove to people the processes are simple, they can be this simple, show people the way. It’s kind of impossible to do that as a single vendor with one tool, so the partnerships are really valuable to the whole field and I think we’ll see more of that in 2024.

This interview was produced in partnership with Bio-Techne.