Patricia Inácio, PhD, science writer —

EIP Pharma is launching a proof-of-concept Phase 2 clinical trial to assess the ability of therapeutic candidate neflamapimod to treat cognitive dysfunction in patients with early-stage Huntington’s disease. The trial (NCT03980938), which is currently recruiting, is taking place at the John Van Geest Centre for…

Emerald Health Pharmaceuticals announced that two of its cannabinoid-derived candidates — CBGA-Q and CBGA-Q-Na Salt — showed anti-inflammatory and neuroprotective effects in a mouse model of Huntington’s disease. However, these benefits were not superior to those obtained from treatment with the company’s already patented EHP-102 compound. The…

RG6042, formerly called IONIS-HTTRx, a potential therapy for Huntington’s disease, was able to reduce the levels of mutant huntingtin (mHTT) protein in early-stage patients, according to results from a Phase 1/2 clinical trial. Trial results were published in the study, “Targeting Huntingtin Expression in Patients with…

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…