Having 35 copies of the CAG triplet in the gene that causes Huntington’s disease is not a problem. Inheriting 40 could be a sign that goes unnoticed for decades, until reaching 80. From there, the process accelerates and neural death occurs when reaching 150 repeats. Huntington’s disease neurodegeneration is not determined by what, but by how much, according to a study conducted at the Broad Institute.
Scientists at Harvard Medical School have shown that in mice lacking amyloid beta (Aβ), the fundamental hallmark of Alzheimer's disease (AD), neurons died from the effect of the most harmful mutation of this neurodegenerative disease. They showed that presenilin (PS) could be behind the origin of the disease without the need for Aβ. They maintain that it is time to update theories and redirect efforts.
Big pharma continues to invest heavily into molecular glue degraders. The latest deal has Eisai Co. Ltd. striking up a research collaboration that could bring Seed Therapeutics Inc. up to $1.5 billion in up-front and milestone payments. The two said they plan to discover, develop and commercialize MGDs for several undisclosed neurodegeneration and oncology targets.
Big pharma continues to invest heavily into molecular glue degraders. The latest deal has Eisai Co. Ltd. striking up a research collaboration that could bring Seed Therapeutics Inc. up to $1.5 billion in up-front and milestone payments. The two said they plan to discover, develop and commercialize MGDs for several undisclosed neurodegeneration and oncology targets.
Alzheon Inc. has raised $100 million in a series E financing round to push its oral drug candidate for early Alzheimer’s disease (AD), ALZ-801 (valiltramiprosate), through a late-stage, Apolloe4 study.
At the 2024 Alzheimer’s & Parkinson’s Diseases Conference this week, Michel Goedert from the MRC Laboratory of Molecular Biology in Cambridge gave the Donald L. Price Memorial Plenary Lecture entitled, “Cryo-EM structures of disease filaments from human brains.” Donald Price was particularly well-known for his work on plaques and tangles in nonhuman aged primate brains and for the development of several animal models of neurodegenerative diseases.
Researchers from JCR Pharmaceuticals Co. Ltd. have presented new data for JR-171, a novel enzyme replacement therapy currently in early clinical development for the treatment of mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome.
Researchers from Indiana University are seeking patent protection for an electromagnetic field (EMF) generation system for treating neurodegenerative diseases. The EMF generation system emulates a small-scale magnetic resonance imaging (MRI) machine, producing the same 64 MHz frequency at a much lower operating power.
Subcommisural organ (SCO)-spondin is a brain-specific glycoprotein essential for neurogenesis with a high impact on neuronal development; thus, reduction in the production of SCO-spondin may lead to a lack of regeneration in neurological disorders.
Using microglia and an unbiased screening method, investigators have identified almost 60 previously unknown targets for γ-secretase. Investigators from KU Leuven and colleagues published their results in Molecular Cell on Nov. 16, 2023.