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Home » Huntington’s disease

Articles Tagged with ''Huntington’s disease''

Art concept for gene therapy research

Voyager sets sail once again with Novartis in $1.3B gene therapy pact

Jan. 2, 2024
By Karen Carey
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.
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DNA in drug capsules
Neurology/Psychiatric

Voyager collaborates with Novartis on gene therapies for Huntington’s disease and spinal muscular atrophy

Jan. 2, 2024
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).
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With more phase I/II data, Uniqure ‘paves road while walking’ in HD gene therapy

Dec. 19, 2023
By Randy Osborne
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.
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Dorsal striatum and its neurons in Huntington's disease
Neurology/Psychiatric

SfN 2023: Lessons from Huntington’s successes, and failures

Nov. 13, 2023
By Anette Breindl
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.”
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Neurology/Psychiatric

Gene editing approaches for the treatment of Huntington’s disease and ALS by Huidagene Therapeutics

Oct. 26, 2023
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.
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Neurology/Psychiatric

Design Therapeutics divulges new compounds for treatment of Huntington’s disease

Oct. 24, 2023
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.
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Illustration comparing healthy vs. Huntington's disease neuron and brain
Neurology/Psychiatric

Skyhawk’s RNA splicing modifier for Huntington’s disease cleared for Australian trial

Oct. 24, 2023
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.
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Neurocrine’s Ingrezza gets expanded FDA nod in chorea associated with HD

Aug. 21, 2023
By Jennifer Boggs
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.
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Brain cells in the hippocampus under microscope.
Neurology/Psychiatric

Glial cell sibling rivalry could be useful for brain repair

July 26, 2023
By Anette Breindl
Transplanted human glial cells could outcompete human glia in a chimeric mouse model of Huntington’s disease, inducing apoptosis. And younger health cells could outcompete older ones. The findings, which appeared online in Nature Biotechnology on July 17, 2023, help pave the way for testing glial cell transplantation as a therapeutic strategy in neurodegenerative disorders.
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Neurology/Psychiatric

Remix Therapeutics patents new RNA splicing modulators

June 22, 2023
Remix Therapeutics has disclosed compounds acting as RNA splicing modulators reported to be useful for the treatment of cancer and Huntington’s disease.
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