We are a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease. Founded by Professor Jan Smeitink, a world-leader in mitochondrial medicine, we are advancing our proprietary science through a wholly-owned clinical and preclinical small molecule pipeline of potential medicines.
Our priority is to rapidly develop our pipeline to deliver transformative medicines for patients living with mitochondrial disease. Our in-house scientists are driving innovative research projects and building a portfolio of promising compounds. We have active discovery programmes underway developing new therapies, biomarkers, diagnostic applications and new read-out technologies in the field of mitochondrial diseases.
Our lead pipeline asset, KH176, is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. It has been granted Orphan Drug Designation for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease in Europe and for all inherited mitochondrial respiratory chain disorders in the US.
We work in collaboration with patient organisations internationally as well as a global clinical and academic network to accelerate the discovery and development of our potential medicines for patients with mitochondrial diseases
Our priority is to rapidly develop our pipeline to deliver transformative medicines for patients living with mitochondrial disease. Our in-house scientists are driving innovative research projects and building a portfolio of promising compounds. We have active discovery programmes underway developing new therapies, biomarkers, diagnostic applications and new read-out technologies in the field of mitochondrial diseases.
Our lead pipeline asset, KH176, is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases. It has been granted Orphan Drug Designation for MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders and Leigh disease in Europe and for all inherited mitochondrial respiratory chain disorders in the US.
We work in collaboration with patient organisations internationally as well as a global clinical and academic network to accelerate the discovery and development of our potential medicines for patients with mitochondrial diseases
Location: Netherlands, Gelderland, Nijmegen
Employees: 11-50
Total raised: $5.65M
Founded date: 2012
Funding Rounds 1
| Date | Series | Amount | Investors |
| 06.05.2025 | - | $5.65M | - |
Mentions in press and media 6
| Date | Title | Description |
| 14.07.2025 | Pretzel Therapeutics: Investment Raised From The Mito Fund | The United Mitochondrial Disease Foundation’s venture philanthropy arm, The Mito Fund, has invested in Pretzel Therapeutics, a Massachusetts-based company with research facilities in Mölndal, Sweden. Pretzel is focused on using cellular ene... |
| 11.07.2025 | The Mito Fund Announces Investment in Pretzel Therapeutics | The United Mitochondrial Disease Foundation's venture philanthropy arm has invested in Pretzel Therapeutics, a company working on novel treatments for POLG and mtDNA depletion related mitochondrial diseases. PITTSBURGH, July 11, 2025 /PRNew... |
| 06.05.2025 | Khondrion Secures Up To €5M | Khondrion, a Nijmegen, the Netherlands-based clinical-stage biopharmaceutical company developing therapies for primary mitochondrial disease (PMD), received an Innovation Credit of up to €5M from the Netherlands Enterprise Agency. This gove... |
| 03.06.2022 | Time Passages: Bruce Goldsmith out at Jim Wilson's gene therapy biotech; Flagship reveals Michael Severino’s CEO gig | Bruce Goldsmith → When Passage Bio readjusted its pipeline priorities, pruned its workforce by 13% and saw its R&D chief Eliseo Salinas walk out in March, it would be natural to assume that this confluence of events could ... |
| 14.08.2013 | Foundations team up to fund translational research on mitochondrial diseases, Alzheimer’s | Pittsburgh’s Knopp Biosciences, Silicon Valley’s Edison Pharmaceuticals and Khondrion in The Netherlands are a few companies that are developing drugs for these conditions. Areas of priority for the grants include discovery and development ... |
| - | Foundations team up to fund translational research on mitochondrial diseases, Alzheimer’s | Two foundations are pairing up to fund work toward new treatments for a group of diseases caused by mitochondrial dysfunction. The Alzheimer’s Drug Discovery Foundation and Foundation for Mitochondrial Medicine will award up to $200,000 to ... |