- The goal of the collaboration is to find new drugs for a rare metabolic disorder that causes severe lung and liver symptoms.
- Kuure and her team test the drug molecules of Gain Therapeutics using mouse models they have developed.
- In the best case, the investigated drugs could then proceed to clinical trials.
Better preclinical models are needed to increase the effectiveness of new drug development for diseases, believes Satu Kuure, Docent of Developmental Biology at the University of Helsinki, Finland. According to her, a great deal of drug testing is still conducted with models that do not follow the course of the disease in humans. That is why animal tests often provide promising results, but when tested on humans, the medicine only works for a few.
“This leads to a situation where the drug or treatment that has been developed cannot be advanced”, Kuure states.
Kuure and her research group have lengthy experience in developing disease models. Now Kuure has joined forces with Swiss company Gain Therapeutics that wants to bring patients new treatments for the metabolic disorder caused by Alpha-1 Antitrypsin Deficiency. In Finland, approximately 300,000 people carry the genetic defect causing it. However, the onset of the disease requires several unlucky coincidences.
“The main symptom is pulmonary insufficiency similar to COPD, which appears in the carrier of the genetic mutation as a young adult”, Kuure describes.
I can critically evaluate whether we can use the mouse model we have developed for testing a new drug compound.
Gain Therapeutics is always looking for certain scientific expertise for its projects. In the three-year Caserpina collaboration, the goal is to create a mouse model, which, in addition to pulmonary insufficiency, also captures the liver symptoms associated with Alpha-1 Antitrypsin Deficiency. A certain group of patients suffers from this, and no treatment is available yet.
“We hope that we can develop a treatment for the liver disease along with improving the treatment options for the lung disease”, says Joanne Taylor, the company’s Senior Vice President of Research.
Kuure and her team were asked to join the project because they know how to create such a dual model. At the University of Helsinki, they have studied the developmental biological mechanisms associated with rare diseases of Finnish inheritance. Kuure has also led the FinnDisMice project that focuses on disease inheritance modelling.
A good animal model replicates the human disease as accurately as possible. This helps the company minimize the risks of drug development and bring medicines to patients more quickly.
“The more customized and precise the model is, the clearer the results we can expect”, Taylor states.
Gain Therapeutics focuses on finding new drugs for difficult-to-treat diseases. Drug testing tools developed by researchers are important to the company because similar models cannot usually be found on the commercial market.
“As we work with rare diseases, not many preclinical tools are at our disposal. It’s through academic collaborations, such as this, that we are able to improve on what’s available to us and to the field as a whole.”
In the Caserpina project, Kuure and her team produce a mouse model with Alpha-1 Antitrypsin Deficiency, in which promising drug molecules are tested. Before this, the other collaborators do their part; the Institute for Research in Biomedicine studies the effects of the drug molecules in cell models, after which the activity of the molecules is tested in Newcell Biotech’s mini lungs.
Kuure is participating in this kind of project for the first time. She is impressed with how effectively the cooperation is planned and monitored. Peeking into the business world and networking have been inspiring experiences.
“From my point of view, it has been very developing”, Kuure states.
An important role in the project is played by the Laboratory Animal Center of the University of Helsinki. Gain Therapeutics is happy with the University’s high level of scientific competence along with the smooth cooperation in administrative and legal matters.
“Given the great interaction that we have experienced with Satu and the University of Helsinki, we hope that this collaboration will be the start of a lasting relationship, long after the grant period has come to an end”, Taylor says.
The Caserpina project is funded by the Eurostars programme of 37 countries, which helps small and medium-sized companies create new innovative products. In Finland, Eurostars funding is distributed by Business Finland.
The results of the project will be ready by spring 2026. The large goal is to have a new treatment available in the future for at least some patients suffering from Alpha-1 Antitrypsin Deficiency.
“It is our dream that one of these drug molecules can be taken to the clinic”, Kuure summarizes.
Contact us and we will design the project according to your individual needs: business@helsinki.fi