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In recent years, stem cell-based cell therapies, which involve the transfer of stem cells or their derivatives into a patient for therapeutic purposes, have provided new hope for patients suffering from incurable diseases. However, despite major advances in stem cell research, many promising candidates never make their way to clinical trials.
Reasons for this gap between research and the clinic include strict regulatory requirements that vary between different countries and a lack of facilities that can manufacture large, consistent quantities of cell therapy products.
Technology Networks spoke with Dr. Bo Wiinberg, chief business development officer at the Novo Nordisk Foundation Cellerator, to learn more about the current obstacles halting advances in stem cell-based cell therapies and what can be done to overcome them. Wiinberg also discusses how the Novo Nordisk Foundation Cellerator is designed to help further develop cell therapies that have already been successfully tested in animals and manufacture these for early clinical trials.
Blake Forman (BF):
Senior Science Writer
Technology Networks
Blake pens and edits breaking news, articles and features on a broad range of scientific topics with a focus on drug discovery and biopharma. He earned an honors degree in chemistry from the University of Surrey. Blake also holds an MSc in chemistry from the University of Southampton. His research project focused on the synthesis of novel fluorescent dyes often used as chemical/bio-sensors and as photosensitizers in photodynamic therapy.
Can you discuss the obstacles in translating successful stem cell research into clinical trials?
Chief Business Development Officer
Novo Nordisk Foundation Cellerator
Dr. Bo Wiinberg is a seasoned leader with extensive experience from both academia, the pharmaceutical and biotech sectors, focusing on strategic and operational leadership throughout the drug development value chain. Wiinberg was previously an entrepreneur in residence at the Bio Innovation Institute and at Novo Nordisk A/S, he held several senior positions over the years, including head of strategy and operations for the Stem Cell unit. Wiinberg is a doctor of veterinary medicine, earned a PhD from the University of Copenhagen and has numerous publications in peer-reviewed journals. Additionally, he has participated in executive education programs in corporate governance and business leadership.
When we were developing a pipeline of stem cell-based cell therapies at Novo Nordisk, a lot of what we did was scout existing projects that we could help accelerate and bring to the clinic. We did this with a few projects at the academic stage and it was very apparent that technically there was a lot that had to be altered to get these projects into clinical trials. For example, the researchers might not have chosen a stem cell line that, from a regulatory point of view, would be suitable for a clinical trial. When you must change a factor like that it can take years and cost millions of dollars. Successful stem cell-based therapy development is about making the right choices early in the process development because it’s hard to make changes to these processes retrospectively.
It's also important to look beyond the first human dose, which is a classic way to approach small molecule and biologic development because you can tweak the process based on learnings from phase 1 trials. However, this is very difficult when developing stem cell therapies. It’s important from the beginning of your development process to consider the result, for example, is the process scalable? Is the technology used in development transferable to manufacture? If you get these initial steps wrong, changing things further along the process is almost prohibitively expensive.
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Finally, licensing the cell lines and key technologies used to develop the therapy is paramount. If you are a startup and have never gone through these processes before, it can be extremely complex to navigate and get right the first time.
Senior Science Writer
Technology Networks
Blake pens and edits breaking news, articles and features on a broad range of scientific topics with a focus on drug discovery and biopharma. He earned an honors degree in chemistry from the University of Surrey. Blake also holds an MSc in chemistry from the University of Southampton. His research project focused on the synthesis of novel fluorescent dyes often used as chemical/bio-sensors and as photosensitizers in photodynamic therapy.
What can be done to help close the gap between cell therapy research and clinical trials?
BW:
Chief Business Development Officer
Novo Nordisk Foundation Cellerator
Dr. Bo Wiinberg is a seasoned leader with extensive experience from both academia, the pharmaceutical and biotech sectors, focusing on strategic and operational leadership throughout the drug development value chain. Wiinberg was previously an entrepreneur in residence at the Bio Innovation Institute and at Novo Nordisk A/S, he held several senior positions over the years, including head of strategy and operations for the Stem Cell unit. Wiinberg is a doctor of veterinary medicine, earned a PhD from the University of Copenhagen and has numerous publications in peer-reviewed journals. Additionally, he has participated in executive education programs in corporate governance and business leadership.
To close the gap between research and clinical trials, it's all about detailed planning, having a clear understanding of the end goal, the target product profile and having the expertise and technology in place to execute efficiently. It's, as an example, not just about choosing the technology that's available in the lab that you've been working in, but understanding what alternatives are available and making an informed choice about which one is best, both for developing the product and bringing it all the way to patients, ensuring both approval and broad access.
BF:
Senior Science Writer
Technology Networks
Blake pens and edits breaking news, articles and features on a broad range of scientific topics with a focus on drug discovery and biopharma. He earned an honors degree in chemistry from the University of Surrey. Blake also holds an MSc in chemistry from the University of Southampton. His research project focused on the synthesis of novel fluorescent dyes often used as chemical/bio-sensors and as photosensitizers in photodynamic therapy.
Can you tell us more about the Novo Nordisk Foundation Cellerator and how this initiative is positioned to help close this gap?
BW:
Chief Business Development Officer
Novo Nordisk Foundation Cellerator
Dr. Bo Wiinberg is a seasoned leader with extensive experience from both academia, the pharmaceutical and biotech sectors, focusing on strategic and operational leadership throughout the drug development value chain. Wiinberg was previously an entrepreneur in residence at the Bio Innovation Institute and at Novo Nordisk A/S, he held several senior positions over the years, including head of strategy and operations for the Stem Cell unit. Wiinberg is a doctor of veterinary medicine, earned a PhD from the University of Copenhagen and has numerous publications in peer-reviewed journals. Additionally, he has participated in executive education programs in corporate governance and business leadership.
What we are building is a physical facility, which was originally primarily designed for chemistry, manufacturing and controls development and good manufacturing practice manufacturing, because we needed that infrastructure to be available for startups and spinouts in Denmark. However, as the project developed, we realized that process development is one of the main obstacles to getting these therapies to market. We discovered that before you even get to manufacturing, taking a process that academic researchers have developed and may have tested in animals successfully, and transferring this technology to humans was a huge obstacle.
What we want to provide with the Cellerator is access to experts who can hands-on help developers choose the best technologies and identify any critical failures in existing processes that will need to be changed to get regulatory approval.
It’s not just about building the infrastructure but partnering with technology providers and setting up a team of experts who understand the regulatory landscape and what is needed to get to that first human dose and beyond.
Senior Science Writer
Technology Networks
Blake pens and edits breaking news, articles and features on a broad range of scientific topics with a focus on drug discovery and biopharma. He earned an honors degree in chemistry from the University of Surrey. Blake also holds an MSc in chemistry from the University of Southampton. His research project focused on the synthesis of novel fluorescent dyes often used as chemical/bio-sensors and as photosensitizers in photodynamic therapy.
The Cellerator facility will be located at the Technical University of Denmark, could you expand on why this location was chosen?
BW:
Chief Business Development Officer
Novo Nordisk Foundation Cellerator
Dr. Bo Wiinberg is a seasoned leader with extensive experience from both academia, the pharmaceutical and biotech sectors, focusing on strategic and operational leadership throughout the drug development value chain. Wiinberg was previously an entrepreneur in residence at the Bio Innovation Institute and at Novo Nordisk A/S, he held several senior positions over the years, including head of strategy and operations for the Stem Cell unit. Wiinberg is a doctor of veterinary medicine, earned a PhD from the University of Copenhagen and has numerous publications in peer-reviewed journals. Additionally, he has participated in executive education programs in corporate governance and business leadership.
There is a lot of expertise in both mammalian cell and cell therapy manufacturing at the Technical University of Denmark, with a proven track record of developing new technologies that creates valuable collaboration opportunities. In addition, being in a university setting allows us to take advantage of existing knowledge and the workforce. We hope to work with the university to upskill young researchers to help progress the field of cell therapy. Finally, Copenhagen itself, which the facility is just a little bit north of, has a fantastic innovation ecosystem.
BF:
Senior Science Writer
Technology Networks
Blake pens and edits breaking news, articles and features on a broad range of scientific topics with a focus on drug discovery and biopharma. He earned an honors degree in chemistry from the University of Surrey. Blake also holds an MSc in chemistry from the University of Southampton. His research project focused on the synthesis of novel fluorescent dyes often used as chemical/bio-sensors and as photosensitizers in photodynamic therapy.
What impact do you hope the Cellerator initiative will have on the larger cell therapy industry in the next 10 years?
BW:
Chief Business Development Officer
Novo Nordisk Foundation Cellerator
Dr. Bo Wiinberg is a seasoned leader with extensive experience from both academia, the pharmaceutical and biotech sectors, focusing on strategic and operational leadership throughout the drug development value chain. Wiinberg was previously an entrepreneur in residence at the Bio Innovation Institute and at Novo Nordisk A/S, he held several senior positions over the years, including head of strategy and operations for the Stem Cell unit. Wiinberg is a doctor of veterinary medicine, earned a PhD from the University of Copenhagen and has numerous publications in peer-reviewed journals. Additionally, he has participated in executive education programs in corporate governance and business leadership.
Everyone in the field hopes to see more cell therapies reaching patients. To achieve this, we need to generate more data from clinical trials to understand what is biologically feasible to inform the next generation of projects. We hope to see the acceleration of stem cell research into clinical trials and not just in the oncology CAR T space. We also hope to see this acceleration where it’s currently harder to get projects into clinical trials such as with regenerative medicine and allogeneic therapies. In addition, because we are a philanthropic organization we hope to bring together biotechnology companies, academics and pharma, in one big collaborative effort to push this field forward.
Driving innovation is a key goal for us. If you look at where the stem cell field is today compared to small molecules and other biological drugs, it's still in its infancy. The cost and risk associated with stem cell therapy development is a major contributor. Many things must happen on the technological front for the costs to come down significantly for producing these therapies. We're now considering how we can support with soft funding for academic groups. We are also considering what we can do to encourage venture capitalists to engage more in this therapy type and become more risk-willing. Being a trusted partner that can help de-risk the entire development process should help with this aim.
