About Khondrion About Mitochondrial Disease
In-depth understanding of mitochondrial disease
More about R&D
Khondrion has received an Orphan Drug Designation from the European Commission of its frontrunner compound KH176, in the treatment of Leigh syndrome
Within all cells of the human body, mitochondria act as a powerhouse – collectively producing energy that is essential for life. When these mitochondria are defective, the result can take the form of a wide range of serious and debilitating illnesses. Signs and symptoms of mitochondrial diseases can include: fatigue, intolerance to exercise, muscle weakness and loss of muscle coordination, heart failure, diabetes, deafness, blindness, stunted growth, and learning disabilities (see figure below for details). Leigh Disease, MELAS , LHON and Friedreich Ataxia belong to the ever-expanding group of mitochondrial diseases.
Mitochondrial failure is usually the result of genetic defects in either the mitochondrial DNA or the nuclear DNA, but can also be caused by environmental factors, including adverse reactions to certain drugs. While it is possible to alleviate several clinical problems, including diabetes, there is still no cure for mitochondrial disease. That's why at Khondrion, we strive to contribute all we can to finding a cure for mitochondrial diseases.
Clinical signs and symptoms of mitochondrial disease
Khondrion works closely with a number of national and international patient and funding organisations in the area of mitochondrial disease. These organisations not only represent an essential source of knowledge for our company and other stakeholders, but also for patients.
Together with a panel of international experts, we have created a clinical guide on Mitochondrial Medicine. The illustrated booklet provides an extensive overview of mitochondrial disease, and can be ordered.
Khondrion primary focuses on the development of therapies for inherited mitochondrial diseases, including Leigh Disease, MELAS, Leber’s Hereditary Optic Neuropathy and other Respiratory Chain / Oxidative Phosphorylation disorders. Our company's focus is on our own product development programmes. We have extensive experience in mitochondrial medicine, unique quantitative live-cell imaging technologies and in-depth, characterised human cell panels. Moreover, we have also developed a portfolio of pre-clinical drug candidates that are in the process of entering clinical phase. Fully operational since 2012 and located in Nijmegen, The Netherlands, our company benefits from an exclusive collaboration with the Nijmegen Center for Mitochondrial Disorders (NCMD) at the Radboud University Nijmegen Medical Center and national and international Foundations. The NCMD is internationally known as a leading full-facility expert center for clinical care, biochemical, pathological and molecular diagnostics and research on patients with disturbances to their mitochondrial energy metabolism. These strategic and long-term alliances ensure a close interaction between research, diagnostic, patient facilities, and patient organisations and allows us to maintain a scientific link with other internationally recognised mitochondrial medicine expert centers across the globe.
Khondrion's mission is, based upon our unique expertise, to develop a cure for patients suffering from mitochondrial diseases who currently lack effective treatment options. Based upon scientific quality and ethical principles we will realize our goal in the most efficient way from a development as well as from a financial point of view. We recruit top class staff and they are proud to be part of the Khondrion team.
At Khondrion, our aim is to foster the discovery and development of targeted and highly innovative solutions for people suffering from inherited mitochondrial disease. We believe that the key to doing so not only lies in our in-depth, scientific knowledge of these diseases, but also in our high degree of understanding of the needs of the people they affect.
We are also well aware that to accelerate the discovery and development of potential drug therapies for mitochondrial diseases, continuous and active collaboration with patient organisations, relevant research institutes and pharmaceutical companies is absolutely essential.
Khondrion diligently strives to accomplish the following objectives:
• To develop a cure for mitochondrial diseases
• As a consequence, to develop a strong portfolio of pre-clinical and clinical drug candidates for a variety of mitochondrial diseases
• To provide reliable and up-to-date information to people suffering from mitochondrial diseases, as well as their families.
With more than 25 years experience in patient care, diagnostics and research of patients suffering from mitochondrial disease, Jan is the Founder and CEO of Khondrion. He also holds a position as Professor in Mitochondrial Medicine at the Radboud University Nijmegen Medical Centre (RUN-MC) in The Netherlands and has been appointed foreign adjunct professor at the Karolinska Institutet in Stockholm, Sweden. In addition, he coordinates the Nijmegen Centre for Mitochondrial Disorders and the Centre for Systems Biology and Bioenergetics, at the Radboud UMC. He has published more than 300 peer-reviewed scientific papers.
Julien holds a PhD in molecular and cellular biology with a major in Pharmacology from the University of Strasbourg, France. Prior to working at Khondrion, Julien was researcher at the Centre for Systems Biology and Bioenergetics of the Radboud University Medical Centre, The Netherlands, focusing on the identification of new drug targets for mitochondrial disease. He has expertise in the discovery of drug candidates and their pre-clinical development. Julien is responsible for internal and external research and development programs and is managing the day-to-day operations of the Khondrion Laboratories.
With over 20 years of experience in Drug Innovation and Clinical Research, Edwin became Khondrion’s Chief Medical Officer, responsible for its clinical development programs. Prior to joining Khondrion, Edwin was Clinical Research Director for Kinesis Pharma (leading an early clinical development and translational science group) preceeded by positions with increasing responsibility at latency Organon and Actelion Pharmaceuticals in Switzerland. In his positions, Edwin supervised a large number of clinical programs in a wide range of indication areas, has had extensive contacts with most regulatory agencies, and served as an expert reviewer for a number of investment funds. Edwin is a PharmD and holds registrations as a Clinical Pharmacologist and as a specialist in Pharmaceutical Medicine. Edwin is an invited lecturer Clnical Drug Development at Utrecht University and several commercial drug development courses.
Dennis is Khondrion’s CFO. Dennis also holds a position as Financial Funding Affairs at the Technology Transfer Office at the Radboud University Nijmegen Medical Centre, the Netherlands. He has extensive international experience in financial-, project- and business control as well as financial management of Grants and cost price modeling.
Khondrion's team members have a long-lasting experience in mitochondrial medicine, pre-clinical and clinical drug development.
By alphabetical order:
Julien Beyrath / Lionel Blanchet / Sjenet van Emst / Sarah Foriel / Lisanne Houben / Eligio Iannetti / Werner Koopman / Dennis Lammers / Svetlana Pecheritsyna / Mina Pellegrini / Jan Smeitink / Edwin Spaans / Peter Willems / Peter van Zandvoort
Khondrion is supported by a team of corporate, strategic and legal advisors that complements its expertise and facilitates the work.
We are always looking for talented and enthusiastic people. If you are inspired by what we do and would like to be part of it, please send an open solicitation to firstname.lastname@example.org. If your profile fits our hiring needs, we will contact you to discuss opportunities.
We attach great value to offering a variety of interesting internships and graduation projects to students from a variety of backgrounds. If you’re interested in working in an ambitious environment, contact us at email@example.com.
Khondrion is optimizing its lead compounds which act at various cell biological processes affected by mitochondrial dysfunction. Our company’s corporate development plan is based on our in-depth understanding of the clinical, biochemical and biological consequences of impaired cellular energy metabolism in health and disease. This unique knowledge enables our team to develop superior drug candidates which display novel activity in inherited mitochondrial diseases. These drug candidates are developed in an extremely targeted manner, referred to as precision medicine.
Khondrion is currently performing in-house drug discovery programmes for the development of new therapies, biomarkers, diagnostic applications and new read out technologies in the field of mitochondrial diseases. We have developed our own portfolio of promising compounds using our unique predictive human cell-based diseases models for inherited mitochondrial diseases.
Khondrion’s proprietary technology platform lies on cell-based disease models built upon a panel of primary human cell lines with different degrees of impairment in their energy production system. We have developed our own portfolio of promising compounds using our unique predictive human cell-based disease models for inherited mitochondrial diseases.
In addition to target discovery and lead optimisation, the models allow for mitochondrial safety profiling during lead development, as well as for the identification of compounds which display the required efficacy profile but not the adverse mitochondrial effects.
Khondrion attaches great importance to sustain creativity, innovation and education to consolidate the future development of targeted solutions for patients suffering mitochondrial disease.
In this context, Khondrion coordinates and participates to several European research and educational initiatives in collaboration with academic and/or industrial partners.
Eligio Iannetti, Peter Willems, Mina Pellegrini, Julien Beyrath, Jan Smeitink, Lionel Blanchet, Werner Koopman.
The International Journal of Biochemistry and Cell Biology, 63 (2015) 66-70.
Sarah Foriel, Peter Willems, Jan Smeitink, Annette Schenck, Julien Beyrath.
The International Journal of Biochemistry and Cell Biology, 63 (2015) 60-65.
Lionel Blanchet, Jan A.M. Smeitink, Sjenet E. van Emst-de Vries, Caroline Vogels, Mina Pellegrini, An I. Jonckheere, Richard J.T. Rodenburg, Lutgarde M.C. Buydens, Julien Beyrath, Peter H.G.M. Willems and Werner J.H. Koopman.
Scientific Reports, 26;5:8035 (2015)
Gerald Pfeffer, Rita Horvath, Thomas Klopstock, Vamsi K. Mootha, Anu Suomalainen, Saskia Koene, Michio Hirano,Massimo Zeviani, Laurence A. Bindoff, Patrick Yu-Wai-Man, Michael Hanna, Valerio Carelli, Robert McFarland, Kari Majamaa, Douglas M. Turnbull, Jan Smeitink and Patrick F.Chinnery
Nature Review Neurology 9, 474–481 (2013).
Koopman WJ, Willems PH, Smeitink JA.
New England Journal of Medicine. 366(12):1132-41 (2012).
Koene S, de Laat P, van Tienoven DH, Vriens D, Brandt AM, Sweep FC, Rodenburg RJ, Donders AR, Janssen MC, Smeitink JA.
Developmental Medicine & Child Neurology. 55(8): 698-706 (2013).
Neurology. 83(2):125-33 (2014).
Khondrion Announces Start of Phase 1 Clinical Trial for Mitochondrial Disease
Khondrion appoints Edwin Spaans as Chief Medical Officer
Khondrion has received an Orphan Drug Designation from the FDA of its frontrunner compound KH176, in the treatment of inherited mitochondrial respiratory chain diseases
Khondrion will present its mitochondrial drug development strategy at the conference Innovation for Health in Amsterdam, The Netherlands on February 5th 2015.
Khondrion will give a lecture on Mitochondrial Medicine at the Paediatric Neurology Association of Hong Kong Annual Scientific Meeting held on November 28th 2014.
Khondrion has received an Orphan Drug Designation from the European Commission of its frontrunner compound KH176, in the treatment of Leigh syndrome (EU/3/14/1336)
Khondrion will present its mitochondrial drug development strategy at the AussieMit conference in Perth, Australia on December 1-3rd 2014.
Phone: +31 24 361 06 39E-mail: firstname.lastname@example.org
PO BOX 91016500 HB NijmegenThe Netherlands
Philips van Leydenlaan 15 (west entrance)6525 EX NijmegenThe NetherlandsInternal route number 427