Scaling Platform Delivery Of Gene Therapies With NewBiologix's Igor Fisch

Gene therapies aim to treat the root causes of disease rather than merely the symptoms. In this episode of Supplier Horizons, host Tom von Gunden talks with CEO Igor Fisch of NewBiologix, a developer of AAV-based gene therapy platform technologies. They discuss various target indications for gene therapies, the challenge of manufacturing AAV particles at commercial scale, and supporting biopharma companies in the development and delivery of cost-effective personalized medicines.

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Episode Transcript

Tom von Gunden, Chief Editor, Drug Delivery Leader:

Welcome to another episode of Supplier Horizons, the series in which we hear from leading innovators and thinkers in the supplier to drug delivery space. My name is Tom von Gunden, Chief Editor of Drug Delivery Leader and your host for this series. For today's conversation, I am joined by Igor Fisch, CEO of NewBiologix, which is a platform development company working on enabling cell and gene therapies.

Welcome, Igor.

Igor Fisch, CEO, NewBiologix:

Thank you, Tom. Very nice of you to welcome me, and I'm really happy to have a chat with you today.

Well, it's my pleasure. Thanks for joining us. So, let's get started. I know that NewBiologix is working on platform development in the cell and gene therapy space. Can you start by painting the current landscape of some of the challenges in delivering cell and gene therapies when it comes to the production of cell lines and particles and platforms to do that work?

Yes. So basically, I think the world of biologics took off at the beginning of 2000, so in this century. And we saw the advent of monoclonal antibodies [mAbs]. And today monoclonal antibodies are new biologics that have made the path towards success.

And now what we see happening is new biologics -- and they are usually called ATMPs, or advanced therapy medicinal products -- and these move toward a more personalized medicine. And when I say personalized medicine, that means that the current process and the future process will change and will be more specific for patients. And we see in ATMPs or cell and gene therapy more systematic treatment that will cure the root of the disease and not only the symptoms of the disease.

And today, cell and gene therapies have made huge progress. And NewBiologix is today focusing on gene therapy and, more specifically, into the production of AAV [adeno-associated virus] particles as gene delivery tools to cure diseases.

Great. Well, thanks for that overview. And feel free now to go ahead and dive a little deeper into the science of what you're working on from a development standpoint, and what will go into the platforms that you'll roll out for the folks that are trying to develop cell and gene therapies.

So basically, today in gene therapy, and as I said, specifically into the production of AAV particles, recombinant adeno-associated viruses, the issue is how can we have a treatment to patients that costs less than $3 million to $5 million (US) per patient? And one of the major issues is not only the market segment, which is a rare disease or small patient population, but also the way we should start producing those drugs.

And today, the method of choice to produce those drugs is very heterogeneous. It's called transient transfection. It's extremely expensive because you have a component that is bacterial and a component that is cell biology. So, mixing both creates regulatory issues. So today, what we are trying to sort out at NewBiologix is our ability to improve these platforms that ultimately will have an impact on the cost of goods for those drugs.

Great. And it may be a range, and I understand that, but could you talk a little bit about any of the particular, first up, therapeutic areas or patient targets that these advances may address?

Today, most of the indications for gene therapy AAV particles are basically either ocular or brain or even muscle. And you have to realize that companies [target] these indications because of so-called immune privilege [since] you don't have the usual immune system defense in those different indications.

You have a poor, I would say, a difficult way to generate those particles in terms of production to target those difficult or rare diseases. Now if we want these drugs to one day be important not only in those indications but also in, let's say, cancer oncology, then we need to put in place a process by which you can virtually produce them in a homogenous way, in very large volumes.

So basically today, gene therapy involving AAV particles is focused on ocular disease, in brain diseases, Parkinson's, Alzheimer's, and in muscle disease, such as [muscular] dystrophy and things like that.

Gotcha. Well, great. Thanks for the deep dive there. I know that your model is to develop platforms that would be used by biopharmaceutical companies and probably contract manufacturers to develop therapies. Can you tell me a little bit about how those partnerships and relationships will evolve and work?

So, in my past company named Selexis, we used to develop a platform that was also an enabling platform to produce monoclonal antibodies based on CHO cells. And back in the early days of 2000, people were not believing that you would be in a position to produce mAbs with stable cell lines. And then this platform became extremely powerful, with over 150 drugs and clinical trials, and then cell and gene drugs on the market.

So here at NewBiologix, we intend to apply a similar model, where we will put a lot of science in developing a platform that will allow the production of viral particles in a stable way. So that means you can produce those in much larger tanks. And then of course, bring a lot of new drugs in the form of AAV particles into clinical trials with a higher chance of success in Phase 3 because the production doesn't become a limitation. So, you can start addressing very large markets with a robust and stable platform. So, this is what we want to address at NewBiologix.

Excellent, excellent. So, I think we'll finish by taking it up to a high level. You can take a run at this any way that you like. But as we look out over the horizon of patient needs and therapeutic areas being targeted with cell and gene therapies, if these advancements and innovations in platform development take hold, what would you hope that means for the treatment aspects of patient lives?

What I really hope is that one day we can cure a patient, as I said, at the root of the disease. So, let's say you have a disease that comes from, let's say, a genetic mutation. Then what you want is not to treat the secondary effect of that. You want to treat the gene that is modified, that has been mutated.

So, the idea is how can we bring the drug to the patient so that it will replace the mutated genes? So, I think in the future, in the evolution of medicine, with all these innovations, we'll see that a medicine will be tailored to a patient. Of course, the next questions become: What about big pharma? How can they make their [money]? And that's going to be, of course, a debate in the society. But I believe that innovation will bring drugs to patients and will become personalized.

Excellent. Great. Well, Igor, I want to thank you for joining me for this episode of Supplier Horizons and sharing your perspectives. And I also want to thank our audience for joining us here. We'll see you next time.