Unique Drug Candidate for Treatment and Prevention of Nucleotide Triplet-Repeat Diseases
| Reference number | |
| Coordinator | Karolinska Institutet - Department of Laboratory Medicine |
| Funding from Vinnova | SEK 3 000 000 |
| Project duration | June 2019 - August 2021 |
| Status | Completed |
| Venture | Swelife and Medtech4Health - Collaborative Projects for Improved Health |
Important results from the project
The aim has been to develop a unique drug candidate for the treatment of a group of disorders caused by the expansion of repetitive DNA-sequences. Focus has been on Huntington’s Disease and Friedreich’s Ataxia and candidates have been identified. The project has moved closer to the market, even if Covid-19 has delayed commercialization. We have through consultants and discussions with a drug company initiated many activities, including making preparations for establishing a joint company.
Expected long term effects
By comparing different drug candidates, we have been able to select the most efficient oligonucleotides. This has mainly been through work with different cells derived from patients. We have also been able to strengthen the concept by repeating the effect of treatment in different biological systems. The commercial part has been strengthened through new developments. We have a better picture of what a product may look like and what is needed to take it to the market.
Approach and implementation
The experimental strategy has been to establish relevant and robust assays to enable the analysis of the drug candidates. This has been done through establishing cell lines as well as animal disease models. This has necessitated method development. The commercial part has been developed with the aid of business consultants in order to plan for every step that is needed, including patent strategies. The discussions with the drug company have strengthened this development.