Investor Relations

Dystrogen Gene Therapies is an early stage life sciences company committed to developing therapies for rare genetic neurodegenerative disorders. Our most advance therapy is for Huntington’s disease (HD).

Press Releases:

May 05, 2022New Publication from Dystrogen Gene Therapies Demonstrates that Artificial miRNA Lowers Mutant Huntingtin Level in the YAC128 Model of Huntington's Disease.

The therapy was well tolerated and did not show signs of toxicity during the course of the experiment up to 20 weeks post injection. The treatment reduced the spleen weight to values characteristic of healthy mice and improved motor performance on the static rod test. These data demonstrate that the CAG-targeting strategy and amiRNA have the potential to become a next generation therapeutic for treatment of Huntington's Disease.
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April 16, 2019 Dystrogen Therapeutics Gene Therapy Shows Promise for HuntingtonsRead more
April 05, 2019Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington's DiseaseRead more


20-22 June 2022Krakow, PLEUROBIOTECH - 8th Central European Congress of Life SciencesScientist from Dystrogene presented new research results: "CAG expanded repeats as a good target for gene therapy in Huntington's disease" during the session Gene therapy and Gene editing.
15-18 September 2019Chateau Liblice, Czech RepublicThe 5th Animal Models of Neurodegenerative Diseases,
6-11 July 2019Krakow, PLThe 44th FEBS Congress, Federation of European Biochemical Societies (FEBS)
11-16 June 2019Krakow, PLThe 24th Annual Meeting of the RNA SocietyScientist from Dystrogene presented genetic tools used for silencing the expression of mutated genes containing expanded CAG repeats targeting short hairpin RNAs used to reduce the level of mutant huntingtin. Presented tools may serve as a universal allele – selective reagents for polyQ diseases such as: Huntington’s disease (HD), spinocerebellar ataxias (SCAs) and dentatorubral-pallidoluysian atrophy (DRPLA).

Peer-Reviewed Publications:

May 05, 2022A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington’s disease.Read more
Dec 19, 2019Universal RNAi triggers for the specific inhibition of mutant huntingtin, atrophin-1,
ataxin-3 and ataxin-7 expression.
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Sep 13, 2013Self-duplexing CUG repeats selectively inhibit mutant huntingtin expressionRead more