for Most rare diseases there is no drug approved
There are about 7000 rare diseases, many with known genetic cause, yet only about 5% have a drug that treats them. At the current rate of drug approval, it could take hundreds of years to identify a treatment for many of these diseases.
This process is too slow.
Now is the time to change this.
Patients play ONLY a limited role in drug discovery
While scientists work on discovering new medicines, patients are told to wait (patiently) for a treatment to be approved. For many of these rare diseases, there might not even be any scientists looking for new medicines while the patients wait.
This system doesn’t work.
Now is the time to change this.
Institute for Orphan Drug Discovery
We are a team of scientists that have worked at universities and pharmaceutical companies and who are tired of waiting. We have met many patients with rare genetic diseases and have fought together with their families to run research projects to find new medicines. We believe that patients and scientists should sit around the table as part of the same team.
That’s why we created the Institute for Orphan Drug Discovery. Our mission is to help you find these medicines.
Created by impatient scientists
Most rare diseases have a genetic cause. We make zebrafish models of human diseases by introducing in their DNA the same changes that lead to diseases in patients.
Using these models, we have helped doctors confirm if a new mutation is causing a disease in a child by introducing the same genetic change in zebrafish and looking at the symptoms that the fish develops. We have also helped academic scientists and companies test new drugs for rare diseases by creating zebrafish disease models and using them for screening many molecules at a scale that cannot be done with mice.
More recently we have started working directly with patient families or patient organizations that don’t want to wait for a pharmaceutical company to get interested in their disease.
We don’t want to wait either.
Ready to start a project?
The Institute for Orphan Drug Discovery is a non-profit research organization based in Bremen, Germany, and operates through a network of collaborating laboratories.