Platform Approaches To Personalized Medicines With NanoVation's Dominik Witzigmann

The goal of moving outside the liver to deliver personalized medicines for rare and genetic diseases brings a host of innovation challenges and opportunities. In this episode of Supplier Horizons, host Tom von Gunden talks with CEO Dominik Witzigmann of LNP (lipid nanoparticle) platform developer NanoVation Therapeutics about addressing a wide range of patient needs while partnering with biopharma and navigating the regulatory landscape to do so.

Interested in joining Tom on an episode? Contact him today.

Episode Transcript

Tom von Gunden, Chief Editor, Drug Delivery Leader:

Welcome to another episode of Supplier Horizons, the series in which we hear about innovations in drug delivery from leading providers to the industry. My name is Tom von Gunden, Chief Editor of Drug Delivery Leader and your host for this series. Today I am joined by Dominik Witzigmann, CEO of NanoVation Therapeutics, an LNP platform developer.

Welcome, Dominik.

Dominik Witzigmann, CEO, NanoVation Therapeutics:

Thanks, Tom. I really appreciate your introduction and am looking very much forward to talking about this exciting topic.

Great. Well, thanks for joining us; it’s our privilege and our pleasure. So, let's start at a very high level, looking out over the landscape of patient needs and therapeutic areas. As you look out over those patient needs and therapeutic areas, what are some of the key needs and challenges,  opportunities and avenues for innovation?

That's a really good question. Generally, I have to say the genetic medicine field is super exciting. The main genetic medicines at the moment mainly focus around the liver. So, the innovation really focuses on tissues beyond the liver. And this is really where the innovation then also kicks in to innovate on technologies which overcome the current barriers to nucleic acid delivery.

So how can we reach extra-hepatic tissues beyond the liver to treat various diseases in oncology or rare diseases? And it's really about demonstrating improvements over conventional therapies.

As you do that work in an [LNP platform-developing] organization, are you partnering in any way with others in the industry to work on innovative approaches to patient needs?

Yes, and I would say collaboration is the way to innovate. One example is our partnership with Novo Nordisk, where we work really closely on targeting rare and cardiometabolic diseases. And it's about leveraging our expertise as an LNP [lipid nanoparticle] developer and expertise from the analysis on the cardiometabolic and rare disease side to develop our two lead programs, which are focused on base editing, so a curative therapy for specific rare diseases.

So, my answer to your question in terms of innovation pipeline: It’s diseases that are very complex. And to get nucleic acids to the site of action, you need a really strong team on the delivery side, on the disease side, on the application side. Therefore, we strongly believe in, and we as a company strongly favor, the approach to collaboratively work with other companies and, therefore, drive innovation by strong scientific excellence.

Well, that all sounds good. So now, when we think back to the patient needs out there that you're working on and partnering to work on, what are some of the key challenges in moving forward with those innovative approaches, whether that has to do with the platform or the formulation or any component of what you're doing there?

I would start to answer this question on the formulation side and the patient need side. As mentioned at the start, current generic medicines  ̶̶  and mainly RNA therapies  ̶̶  focus on vaccines or the liver. So, one of the delivery challenges the field is really focused on at the moment, as well as at NanoVation, is how do we get nucleic assets to tissues beyond the liver?

Therefore, it's really about developing new types of LNPs which enable effective therapies, but also safe therapies, and provide the specificity needed to enable therapeutic concepts. And the approach we take here is the development of LNPs which are designed and developed for a specific application.

So, we take into consideration the route of administration: Is it IV? Is it intramuscular? Is it local injection? The payload: Is it the RNA? Is it a circular RNA? Is it DNA? As well as the tissue and cell type to target: Is it in the bone marrow? Is it in the blood compartment? Is it a specific immune cell? And therefore, I like to say we do fit-for-purpose LNP development because we really need to consider the target product profile and therefore develop the LNP which is enabling that genetic medicine.

Gotcha, gotcha. So obviously, in the realm of targeting rare diseases – and doing so, often with personalized approaches, personalized treatments ― how do you see the regulatory landscape when it comes to personalized approaches and personalized medicines in terms of working with regulatory agencies to get approvals?

That's a challenging as well as a super exciting question. I’ll start with the excitement. It's that genetic medicines now, for the first time in human history, enable us to cure diseases on a genetic level. And with gene editing, for example, starting with the liver, there are certain genetic diseases where patients have private mutations. So, we actually have the technologies which would enable curative therapies for each and every single patient.

The question is how do we approach this from a regulatory side? And this is the main question here where we need a strong open dialogue between regulators, innovators, [and] pharmaceutical companies, as well as patient organizations. And there has been a lot of movement in the last couple of years. And especially FDA and [former CBER Director] Peter Marks, they drive forward new guidelines and push forward various initiatives.

So, I'm really looking forward to the future. It will be exciting. It's a matter of, how do we structure our landscape to enable a patient benefit at the end?

I share your excitement. And thinking about those patient benefits, Dom, I'm going to finish with just one final question for you for today. And that is, as we look out at the near horizon and beyond in terms of these genetic approaches and personalized approaches and rare disease targeting, if the innovation efforts that you're working on achieve the results and the success that we assume they will, what do you envision the lives and health of the patients would look like once those things come to fruition?

I would start with the patient in mind. This is really what drives us at NanoVation to work collaboratively with our partners for beneficial patient outcomes. And what I often tell my team is that there's nothing more important than patients. So, we always need to keep the patient in mind in our development. And the benefits I would see ― and there are so many different organs in our body and so many different diseases, there are more than 6000 rare diseases known and even more to be discovered ― where we now have the ability to treat, to cure, or to prevent such diseases.

So from an innovation point of view, I'm really excited about the current ongoing project we have on T-cell therapies, on targeting hematopoietic stem cells for rare diseases. Or also the cancer application side, how to treat cancer or how to prevent it using cancer vaccines. So, there's a lot of excitement in the field, and I really hope that we will see a lot of breakthroughs in the next coming years and decades.

I certainly hope so, too. Dom, I want to thank you for joining me to share your perspective on what you're working on there at NanoVation Therapeutics. And I also want to thank our audience for joining us for another episode of Supply Horizons. We'll see you again next time.