UniQure has just published a study in Science Translational Medicine using a Huntingtin (HTT)-lowering therapy. IRBM is delighted to have been able to contribute to this pivotal work and to be cited on the publication.
Huntingtin is the gene that causes Huntington’s and therapies attempting to lower the levels hold the potential to slow down neurogeneration. UniQure’s approach uses recombinant adeno-associated viral vector serotype 5 expressing a microRNA targeting human HTT (rAAV5-miHTT).
The study demonstrates widespread biodistribution, strong and durable efficiency of rAAV5-miHTT in disease-relevant regions in a large brain. These results pave the way to future studies which look to advance treatment options for Huntington’s Disease.