1. Tell us about your role as Chief Early Development Officer at Khondrion.
There are a lot of different aspects to my role. I help to steer the Khondrion lab as we research the science behind our early-stage rare disease pipeline. I am also responsible for managing the publication of the preclinical and clinical data that comes out of our research. I liaise with academic partners and advisors, and apply for the necessary funding, which is crucial to our ongoing research and development. In addition, I monitor our Chemistry, Manufacturing and Controls (CMC) work and manage our work with Contract Research Organizations (CRO) that supply our preclinical trials and experiments.
2. What does a typical day at Khondrion look like for you?
My work as Chief Early Development Officer started in June 2020, in the midst of a global pandemic. Khondrion, like every other organization, has had to make significant changes to the way we work on a day-to-day basis to ensure staff are able to work safely, while still progressing with this important work. Given the COVID-19 restrictions, I work on site at Khondrion 1-2 days a week, and work remotely the rest of the week. The work I do involves many conversations with team members, colleagues, CROs, external scientists, research partners and advisors, and Zoom has been integral in keeping those interactions going. It has been extremely important to continue our research efforts despite the ongoing pandemic, in order to achieve our goal of bringing new treatments for patients with mitochondrial disease.
3. How did you become interested in mitochondrial disease research?
Although I have worked in several different fields, the common denominator has been the translation of biochemical/cell biological disease into treatments. In most of my previous work environments, labs working on mitochondria were close at hand and I learned a lot about these intriguing organelles. When I moved back to the Netherlands from Finland in 2010, I was delighted to join this exciting field of research and the close-knit mitochondrial community
4. What excites you about Khondrion’s early-stage pipeline?
Khondrion puts a lot of effort into science driven development and I am confident that we will bring real treatment options to patients with mitochondrial diseases, where there are currently no treatments at all. The stories of the patients are often heartbreaking, and it is great to be part of the effort to help a group where there is such a high unmet need.
5. What career goals are you hoping to achieve at Khondrion?
Personally, I am grateful to be able to develop and grow my skills across the many aspects of pharma at a small and nimble company like Khondrion. In big pharma I would not be able to be involved in all facets that are now part of my day-to-day work.
6. Tell us about your career before joining Khondrion.
I have been very fortunate to have spent the majority of my career so far working in various roles across academia. After completing my PhD in biochemistry at the University of Amsterdam, I moved to Tampere in Finland to work on a project in cell signaling and virology funded by a Marie Curie fellowship. I then received funding from the Academy of Finland and worked as a junior group leader for a few years.
After I moved back to the Netherlands with my family I joined the Center for Systems Biology and Bioenergetics in Nijmegen as a biochemist/cell biologist working on a computational model for cellular energy production. I then took up a senior-Post Doctorate position in the Translational Metabolic Lab at the Radboud UMC working to establish a definite diagnosis for patients with novel mutations derived from their whole exome sequencing data by employing complementation experiments. After this, I decided to shift gears to industry and started working for Khondrion – first as a scientist and now as Chief Early Development Officer.
7. What do you see as the biggest challenges in the mitochondrial disease field?
With mitochondrial disease, signs, symptoms and the organs involved can differ from one person to another. This not only makes it a challenging disease to diagnose but makes the search for potential therapies extremely complex in terms of deciding which patients to study and what outcomes to measure. Khondrion is working to overcome this challenge with a stepwise trial approach for our lead drug candidate, sonlicromanol, as well as active and ongoing dialogues with regulators so that we can hopefully bring this much needed treatment, and other future treatments, to patients as quickly as possible.
8. How hopeful are you about the future of mitochondrial disease?
There have been huge advances in mitochondrial disease research since I first started working in the field – there are new technologies and tools being developed all the time to help us develop potential new therapies. We have an incredibly dedicated and hardworking team at Khondrion and I am confident that with this passion and commitment, together with the pace of scientific discovery and research, we will achieve a treatment for patients with mitochondrial disease.