NRG Therapeutics Ltd. has been awarded a grant from Target ALS Foundation to support its discovery program for a treatment for amyotrophic lateral sclerosis (ALS).

Mid-Atlantic Biotherapeutics Inc. and Accelero Biostructures have entered into a research collaboration to identify novel USP30 inhibitors for neurological disorders and other indications.

Vivet Therapeutics SAS has announced its gene therapy VTX-806 has been awarded European orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare disorder of bile acid metabolism.

Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

Researchers from the UK Dementia Research Institute at the University of Cambridge have found how to prevent and reverse tau aggregation using target-specific nanobodies. The team holds great expertise in the role of TRIM21 in the tau environment since William McEwan, senior author of the study, first discovered TRIM21 and, a bit later, defined its contribution to tau immunotherapy efficacy.

CTNNB1 syndrome is a neurodevelopmental disorder characterized by intellectual disability, global developmental delay, microcephaly and motor disabilities, among others, caused by pathogenic loss-of-function variants in the CTNNB1 gene, which encodes β-catenin. This syndrome has no treatment option, with only supportive care available. To address this unmet medical need, researchers from the Broad Institute and Tufts University School of Biomedical Sciences have developed a Ctnnb1 germline heterozygote murine model that mimics the human CTNNB1 syndrome.