Evidence from research has pointed to a positive correlation between high glucocorticoid levels and the advancement of Huntington's disease (HD). Researchers from Leiden University Medical Center and Corcept Therapeutics Inc. have reported on the evaluation of CORT-113176 (dazucorilant), a glucocorticoid receptor antagonist, in mouse models of HD.
Mutant huntingtin (HTT) protein is the main cause of the pathological features leading to Huntington's disease (HD) development, a neurological disorder characterized by CAG repeat expansion in exon 1 of the HTT gene, which causes neuronal dysfunction and death along the brain.
Nearly two years after Novartis AG signed a $1.7 billion deal for options to Voyager Therapeutics Inc.’s adeno-associated virus capsids for central nervous system disorders, the Basel, Switzerland-based company secured rights to develop gene therapies for Huntington’s disease and spinal muscular atrophy in a licensing agreement potentially worth $1.3 billion.
Voyager Therapeutics Inc. has entered into a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG, to advance potential gene therapies for Huntington’s disease and spinal muscular atrophy (SMA).
On-again, off-again investor enthusiasm for Uniqure NV’s Huntington’s disease (HD) gene therapy AMT-130 got another boost as the company followed this summer’s news from phase I/II trials with additional interim data. Shares of Uniqure (NASDAQ:QURE) closed Dec. 19 at $6.64, down $1.34, or 17%, as the company offered results on up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European experiment.
The gene for Huntington’s disease “was cloned in 1993, and everyone thought there was going to be a treatment right around the corner,” Sarah Tabrizi told the audience at the 2023 Annual Meeting of the Society for Neuroscience. Then, “it took 25 years for the first trial targeting the Huntington gene.”
Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.
Research at Design Therapeutics Inc. has led to the discovery of conjugates consisting of a DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to a protein binding moiety through oligomeric backbone linker. They are transcription modulators reported to be useful for the treatment of Huntington’s disease.
Skyhawk Therapeutics Inc. has obtained Australian human research ethic committee (HREC) approval to conduct a phase I trial of SKY-0515 for Huntington’s disease.
The impact of a black box warning on Neurocrine Biosciences Inc.’s expanded U.S. label for Ingrezza (valbenazine) to treat chorea associated with Huntington’s disease (HD) appears up for debate, following FDA approval late Aug. 18. But analysts agree that it is unlikely to affect sales of the VMAT2 inhibitor in tardive dyskinesia, for which the company recently raised 2023 guidance as high as $1.82 billion.
