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.
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.
Remix Therapeutics has disclosed compounds acting as RNA splicing modulators reported to be useful for the treatment of cancer and Huntington’s disease.
Shares in Uniqure NV fell sharply on inconclusive interim data from a U.S. phase I/II trial of its gene therapy for Huntington’s disease. The stock (NASDAQ:QURE) hit a six-year low during trading on June 21, ending the day at $11.62, down 40%.
Researchers from Seelos Therapeutics Inc. presented the discovery and preclinical evaluation of a gene therapy candidate, SLS-009, for the treatment of Huntington’s disease (HD).
One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier.
