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Unique Drug Candidate for Treatment and Prevention of Huntington’s Disease

Reference number
Coordinator Karolinska Institutet - Department of Laboratory Medicine
Funding from Vinnova SEK 1 000 000
Project duration June 2018 - June 2019
Status Completed

Purpose and goal

The project’s aim is to develop a treatment for Huntington’s disease (HD) through the use of our technology, which is based on oligonucleotides, i.e. short sequences of synthetic nucleotides, which directly bind to the disease gene. The treatment is expected to delay disease development. There are two major strategies, industrial development of the treatment procedure in parallel with academic research, where we investigate the effect of chemically modified oligonucleotides on the down-regulation of disease gene expression in different cell models.

Expected effects and result

The focus has been on business development of the innovation as well as lead optimization and validation of our treatment procedure in different disease models. We have an ongoing discussion with several potential collaborative partners from the pharmaceutical industry. At the same time the experimental part has yielded good results in the form of continued development of the method in HD patient cell models. We have validated the treatment procedure and made preparations for its use in HD animal models.

Planned approach and implementation

We have developed an innovative treatment strategy for HD. We have established a biological drug candidate, an oligonucleotide, for which we already have proof-of-concept. This medicine binds directly to the hereditary material, DNA, and blocks the expression of both toxic RNA and protein. We focus on establishing a durable R&D strategy with the ambition to take the product all the way to the market. A strength is that our product is unique in the sense that only our invention enables blocking of toxic RNA and that it also may result in disease prevention.

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

Last updated 6 December 2018

Reference number 2018-00177

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