Uniqure NV shares (NASDAQ:QURE) closed July 9 at $6.67, up $2.89, or 76%, after the firm made public updated interim data including up to 24 months of follow-up findings from 29 treated patients enrolled in the ongoing U.S. and European phase I/II trials of AMT-130 for Huntington’s disease (HD).
A nearly two-year-old partial clinical hold has been lifted by the U.S. FDA on PTC Therapeutics Inc.’s pivotal phase II study in Huntington’s disease. The agency had paused enrollment in October 2022, saying it wanted more data on PTC-518, an orally bioavailable small-molecule splicing modifier, before enrollment could continue.
Philadelphia-based Latus Bio Inc., co-founded by serial biotech entrepreneurs P. Peter Ghoroghchian and Beverly Davidson, launched on May 2 with two lead adeno-associated virus (AAV)-based gene therapy candidates and $54 million in a series A financing.
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).
