Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in the enzyme palmitoyl-protein thioesterase 1 (PPT1) due to biallelic loss-of-function mutations in the gene encoding the lysosomal enzyme, leading to widespread neurological dysfunction and premature death.

Tubulin inhibitors by themselves can limit tumor growth, and they can be particularly effective when conjugated to monoclonal antibodies that target them to tumors. Two such conjugates, trastuzumab emtansine and brentuximab vedotin, have been approved by the FDA. However, many cancers can develop resistance to such inhibitors, highlighting the need for next-generation compounds.

Nabla Bio Inc. has established a new multiyear research collaboration with Takeda Pharmaceutical Co. Ltd. to advance AI-driven design of protein therapeutics.

Recent evidence suggests the gastrin/cholecystokinin-2 receptor (CCK2R) is a potential target for radiolabeled theranostics in cancer, but the stability of minigastrin analogues remains unresolved. Researchers from the National University of Singapore aimed to develop a peptide with improved tumor uptake and prolonged retention in CCK2R+ cancers.

Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.

At the 2025 European College of Neuropsychopharmacology (ECNP) Congress in Amsterdam this week, researchers presented new findings on the role of endocannabinoids in stress, addiction … and the integrity of the blood-brain-barrier.