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.
Aural Analytics Inc. received a breakthrough device designation from the U.S. FDA for its Speech Vitals-ALS technology, a software application that collects and analyzes speech recordings to help monitor amyotrophic lateral sclerosis (ALS) in adults in clinic and home settings. The software could improve management of the devastating disease.
Newco Teitur Trophics ApS has raised €28 million (US$30.1 million) in a series A with which it will lay out a new route to targeting sortilin in the treatment of neurodegenerative diseases. The company is targeting the sortilin-related Vps10p domain containing receptor, which plays a role in regulating a number of pathways involved in the control of neuronal viability and function.
Teitur Trophics ApS, a spinout from Aarhus University seeded by the Bioinnovation Institute in 2020, has completed a €28 million (US$30.1 million) series A financing. Teitur has developed a platform of first-in-class cyclic peptides with a novel mechanism that preserves neuronal function, and these peptides have the potential to treat a broad range of neurodegenerative diseases.