Latus Bio Inc. has launched with a focus on developing novel gene therapy candidates for central nervous system (CNS) disorders. An initial close of $54 million in series A financing will support the company.
After Sage Therapeutics Inc. reported a phase II failure with oral dalzanemdor, also known as SAGE-718, in mild cognitive impairment related to Parkinson’s disease (PD), Wall Street’s eyes turned to ongoing mid-stage efforts with the same N-methyl-D-aspartate receptor-positive allosteric modulator in Huntington’s disease and Alzheimer’s disease.
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.
