Tau is an intrinsically disordered protein that regulates the stability and dynamics of microtubules in physiological conditions. Recent work has revealed the involvement of tau in various neuronal processes. Researchers from the University of California and collaborators aimed to systematically investigate the cellular factors that control the accumulation of tau aggregates in human neurons.
Aquinnah Pharmaceuticals Inc. has disclosed new microtubule-associated protein τ (PHF-τ; MAPT) aggregation inhibitors designed for use in the treatment of Alzheimer’s disease and frontotemporal dementia.
Dewpoint Therapeutics Inc. has announced the selection of a development candidate for its TDP-43 program. The first-in-class small molecule is designed to correct disease-associated TDP-43 condensates, restoring normal TDP-43 function and addressing the core molecular pathology that drives neurodegeneration in amyotrophic lateral sclerosis (ALS) and related diseases.
Aperture Therapeutics Inc. has nominated APRTX-001 as a development candidate, with the program now advancing through IND-enabling studies. APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of frontotemporal dementia and amyotrophic lateral sclerosis, with potential for indication expansion into Alzheimer’s disease.
Transition Bio Inc. and Voyager Therapeutics Inc. have entered into a drug discovery collaboration and license option agreement for novel, selective small molecules for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology.
Neurodegeneration specialist Vesper Bio ApS has announced positive results from the phase Ib/II trial of its oral sortilin inhibitor VES-001 in frontotemporal dementia. The small study involved six participants who had not progressed to symptomatic disease but were carriers of mutations in the GRN gene that codes for progranulin, a growth factor that is essential for neuronal health.
The failure of Alector Inc.’s phase III study of latozinemab in treating dementia halved the company’s stock on Oct. 22. That is also about the same percentage of staff that Alector is letting go after the clinical trial stumble.
Inhibiting histone deacetylase 6 (HDAC6) has therapeutic potential against several neurodegenerative disorders. A collaboration including researchers from Eikonizo Therapeutics Inc., spanning the U.K., U.S. and France, developed EKZ-438, which has shown strong potential against amyotrophic lateral sclerosis and frontotemporal dementia in preclinical studies.
The switch will be flicked today to make the world’s largest dementia-related proteomics dataset freely available to researchers, at the same time as members of the consortium which compiled it publish the proteomics signatures of major neurodegenerative diseases that they uncovered in a first trawl of the data.
Monoclonal antibodies show strong therapeutic potential against neurodegenerative diseases, but a major challenge is ensuring that they can arrive unharmed in the brain at sufficient concentrations. One way around this challenge is to vectorize the monoclonal antibody: encode it within a vector that can infect target cells in the brain and express the antibody in situ.
