Patricia Inácio, PhD,  science writer—

DYST 201, a gene therapy targeting the mutated forms of the gene responsible for Huntington’s disease, has shown promising results, Dystrogen Therapeutics announced. The results will be presented at two meetings in Krakow, Poland — the July 6-11 44th FEBS Congress and the June 11-16 24th Annual Meeting of the RNA Society.

The U.S. Food and Drug Administration (FDA) has given fast track designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. The designation helps to speed the development, testing, and review of therapies for serious diseases with high unmet medical need. “Achieving Fast Track Designation from the…

Genistein, a potent stimulator of cells’ natural “cleaning system” was able to reduce the levels of mutant huntingtin protein in skin cells from Huntington’s disease patients, a study has found. The study, “Genistein induces degradation of mutant huntingtin in fibroblasts from Huntington’s disease patients,” was published in…

The U.S. Food and Drug Administration (FDA) has issued a letter of support encouraging sRNAlytics to further develop its sRNA-FIND platform for discovering and monitoring variants of small RNA molecules that may work as biomarkers for tracking progression of Huntington’s disease decades before symptom onset. In addition, these biomarkers may…

The U.S. Food and Drug Administration has granted orphan drug designation to MP101, an investigational mitochondrial-targeted neuroprotective agent, to treat Huntington’s disease. Mitochon Pharmaceuticals, the therapy’s developer, expects to begin a Phase 1 clinical trial in healthy volunteers in 2019 and move to Phase 2 studies in 2020. “We are…

Several small molecules have been identified by the Alborada Drug Discovery Institute as potential inhibitors to help halt the underlying mechanisms that drive the development of neurodegenerative diseases such as Huntington’s and Parkinson’s disease. Silencing phosphatidylinositol 5-phosphate 4 (PI5P4) kinases has been shown to enhance the natural cleaning system of cells, called autophagy,…

uniQure is launching a Phase 1/2 trial in the United States to evaluate the safety and effectiveness of its Huntington’s disease investigational gene therapy AMT-130. This follows recent approval of an investigational new drug (IND) application for AMT-130 by the U.S. Food and Drug Administration (FDA). “The…

Asuragen has launched a fast, easy-to-use and reliable diagnostic test, called AmplideX PCR/CE HTT Kit, for Huntington’s disease. The AmplideX test is able to detect up to more than 200 CAG trinucleotide repeats within the gene that codes for the huntingtin (HTT) protein. “AmplideX technology continues to demonstrate…

While transplanted fetal nerve cells can survive long term in the brain of a Huntington’s disease (HD) patient, only a specific subset called interneurons thrive, according to a study. Projection neurons — a particular type of nerve cell commonly lost in Huntington’s — were rarely detected, probably due to…

A bioactive compound present in raspberries called salidroside can prevent the cellular toxicity that occurs as a consequence of protein accumulation in certain neurodegenerative diseases, including Huntington’s disease, a study reports. The study, “Identification and microbial production of the raspberry phenol salidroside that is active against Huntington’s disease,” was published…