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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.

The project description has been provided by the project members themselves and the text has not been looked at by our editors.

Last updated 1 October 2021

Reference number 2019-01534