Karin Hoogendoorn is a Board Member at Idogen and renowned expert in the area of pharmaceutical development and regulatory affairs of cell- and gene therapy products or so-called advanced therapy medicinal products (ATMPs). In this interview with BioStock, she shares her thoughts on the current breakthrough of cell therapy in general, as well as the potential challenges and advantages of Idogen’s approach to personalized cell therapy.
Karin, you have many years of experience within the biotech industry and specifically from working with gene- and cell therapy products. What in particular attracted you to join Idogen?
– Yes, I worked for quite a few years at Johnson & Johnson’s pharma company Janssen Biologics in the area of regulatory strategy and CMC*, particularly regarding cell therapy products. I also worked on the development of cell and gene therapy products at Novartis’ headquarters in Basel (e.g., their CAR-T product). What attracted me to Idogen in particular is the company’s innovative tolerogenic dentritic cell technology and unique approach, which I find very exciting.
(*CMC: Chemistry, Manufacturing and Control)
What advantages are you bringing to the Idogen board?
– I bring expertise in CMC development and regulatory affairs from a global perspective. Also, my experience from the twenty years I spent in industry focusing on the CMC development covers many aspects of the manufacturing process and testing of the product, from pre-clinical through clinical development, to the post-approval phase.
What would you say characterizes cell therapies in general, in terms of its market potential and future role as an important weapon in fighting disease?
– Cell therapy is truly personalized medicine where we focus not only on a specific disease but also on the genetic profile of a person when treating patients. In general, I think the therapeutic benefits very much outweigh the possible risks. There are a lot of opportunities in the field of chronic diseases, like for example haemophilia where I think Idogen’s technology can make an important difference and fulfil a significant unmet medical need.
Can you briefly explain the current status of Idogen? What has the company achieved, and what is its focus today?
– The company has already achieved quite a few milestones, e.g., extensive pre-clinical work has been performed in lab experiments and in animals to investigate how the dendritic cell therapy product works in its most advanced indication, haemophilia A patients suffering from immune reaction against the standard treatment with factor VIII. Furthermore, Idogen is in the works to get their production facility certified for the manufacture of the dendritic cells for clinical application. Next to the certification of the production facility, Idogen focuses on the preparation for the first clinical trial in the above mentioned indication, which will be initiated end of 2018.
Which specific factors do you think will lead to a major breakthrough for cell therapy?
– A very favorable benefit/risk ratio, where the efficacy of the product very much outweighs the safety risks; products which really cure diseases, particularily in the area where there aren’t any treatment options available; flexible, closed, and automated production systems, allowing for consistent product manufcature at a few different sites, close to the clinic where the product is administered to the patient.
Idogen is developing new treatments based on the individual patients’ own cells. What would you say are the biggest advantages and challenges of this particular approach to personalized medicine?
– The biggest advantages are from my point of view, that there is a lower risk for immuno rejection as these cells are the patient’s own cells. Hence, the immune system may recognize these cells as “own” and not as “foreign”. Also, the fact that patients’ acceptance to use own cells may be higher than to use of cells from another donor.
– Challenges we are facing in the area of personalized medicine are how to lower the costs of goods, production, and testing costs and also how to develop fully automated and closed manufacturing systems to enable a “one stop shop” production process with in-line testing capabilities.
Idogen is setting up its own production facilities, something which is not so common among small biotech companies. What are the primary advantages of keeping production in-house?
– I think that Idogen is quite unique in this perspective, especially for a small biotech company. It gives great flexibility in terms of planning your R&D, when developing the production process and also when scheduling the GMP manufacturing for clinical trials. In addition, doing the production and testing in-house brings in a lot of hands on experience related to the process and product, which will never be the case when the CMC development and production is sourced out to a contract manufacturing organization. To have the production in-house could also be a unique selling point and a very strong asset when the company enters talks with potential partners.
Big Pharma companies’ pricing policies for novel cell therapies has become somewhat of a hot topic, at least in the US. What is your view on that issue?
– Cell therapy in general is not cheap and it probably never will be. But really – if it can make a difference and let people live longer, better lives with a manageable chronic disease, or if we even can find ways to cure a disease… That would really make a huge difference for the patients. There is also a lot of money to be saved in the longer perspective. Looking at organ transplants or haemophilia, many patients are very young with many years of treatment ahead of them. If we can prevent them from being hospitalised or going back and forth to the hospital for years to be treated, it adds a lot to their quality of life and cuts a lot of costs too. I also think we have to look at changing or improving the reimbursement system for these therapies.
You have contributed to the development of several new gene- and cell therapy products, which specific development milestone are you looking forward to the most in working with Idogen?
– There are several important milestones coming up. If I have to choose one, I must say that I’m really looking forward to the first patient treated, that is a milestone well worth celebrating!