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Interview: Leveraging iPSCs to accelerate drug discovery

By Stefan Braam, PhD

Stefan Braam, PhD has more than a decade of experience in stem cell technology, product development and general management. Over his career, Stefan has published a number of articles in a range of leading scientific journals and is an inventor on multiple patent families. He secured multiple grants and commercial research collaborations and was instrumental in securing pre-seed, seed, Series A and B financing rounds for both Pluriomics and Ncardia.

In this interview, Stefan discusses the challenges and benefits of using real human biology in drug discovery. Plus, learn more about how confidence from investors has led to huge growth in the regenerative medicine field, and what more will be coming in the future.

A few months ago, Ncardia made the news for receiving a US$60 million funding from Kiniciti; what will the funding be used for?

From a technological perspective, Ncardia focuses on the manufacture of cells derived from human induced pluripotent stem cells (iPSCs) in two segments: drug discovery and cell therapy. Human iPSCs are still a relatively new technology in the cell therapy space, but they have garnered considerable interest because of the incredible potential they hold. We have built our iPSC platform to enable development of novel therapeutics, which are subject to additional regulations, so we have been looking for the right partner to help us do that.

Now that we have this funding from Kiniciti, we will be able to build more capacity, including GMP capacity. This will enable us to serve more clients across a wider range, both in terms of therapeutic applications as our platforms mature, and markets.

Ncardia has established itself as a leader in iPSCs. How has the iPSC landscape evolved since we last interviewed you?

It’s absolutely red hot; in the last year we have seen so many successful funding rounds and IPOs for large and small companies. This represents a significant amount of money, and therefore developments, going into the industry, which is very exciting. We have also seen a continuous, rapid uptake of iPSC-derived models accelerating drug discovery. I have been in this field for 15 years and it feels like all the preceding 14 years were needed to bring us to this point.

What has contributed to this huge confidence in this field from investors?

It took time for people to have confidence that this technology might be scalable. Progress has been made in the drug discovery segment and in the development of autologous therapies, which is making a real difference to patients. These have come together in a perfect storm to demonstrate the value of iPSCs.

However, it is relatively early days, so the majority of assets we are seeing are still preclinical. Bringing true human biology into early drug discovery to help with decision-making will reduce pipeline attrition and, as such, wasted expenditure. iPSCs are becoming broadly accepted as a key solution going forward whilst we wait for the enabling technologies, such as closed manufacturing platforms and defined culture media, to catch up in order to bring them to a therapeutic reality.

Now that there’s growing demand in stem cell technologies, what makes Ncardia’s technology stand out?

What we have been able to do, is identify and address bottlenecks from a technological perspective. We started establishing manufacturing processes with scalability in mind from the very first steps, which has enabled us to build a technology platform that is pre-eminent in the market. By leveraging this technology, we have grown an organization that provides a unique service, and where great scientists work collaboratively with our clients.

Can you talk to us about Ncardia’s use of iPSC technology in the field of drug discovery for cardiovascular and the central nervous system (CNS)?

In our field, it is so important to understand the differentiation systems and assays that are available, to help clients overcome challenges and mitigate risks. At Ncardia, we make sure that we get every step in the process exactly right in order to develop the needed disease models: we source our cells from either healthy or diseased donors and leverage our 10+ years of expertise to differentiate them into the required cell type. We then identify and develop the kinds of assays we need to measure the target phenotype, both to identify it and to monitor the impact of a candidate therapeutic.

We have experts who really understand the challenges in drug discovery projects, and we have the technology to solve them; this means we can be very consultative and flexible with our clients to get the best results.

Are there any new platforms or products in the pipeline for Ncardia?

We are rapidly growing at the moment and expect to do more projects in cardio and CNS drug discovery, as well as continuing our service work with clients to boost their disease modelling capabilities. During drug discovery, it’s very important to build large batches of cells so that when screening starts with multiple molecules, the same cells can be used each time for the most consistent results. Therefore, having a consistent supply of cells will enable the most promising results. Having real human models available is incredibly powerful. It enables solving complex questions, such as what genes are influencing my disease phenotype or what compounds can rescue it. We believe iPSC technology is the solution for more effective and efficient drug discovery.

Source: RegMedNet

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