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. Read More
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. Read More
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. Read More
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. Read More
BioWorld Staff Writer Tamra Sami says it’s impossible to talk about changes to the biotech and med-tech landscape in Asia without discussing China, which is eclipsing the region. In the last two decades, China has shifted from imitator to innovator. Read More
Hinge Bio Inc. has obtained IND approval from the FDA to initiate a phase I study of HB-2198 in patients with the B cell-mediated autoimmune diseases systemic lupus erythematosus (SLE) and lupus nephritis. The trial is expected to open this quarter. Read More
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc. Read More
Nabla Bio Inc. has established a new multiyear research collaboration with Takeda Pharmaceutical Co. Ltd. to advance AI-driven design of protein therapeutics. Read More
Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising a Von Hippel-Lindau disease tumor suppressor (VHL)-binding moiety covalently linked to a GTPase KRAS G12D mutant-targeting moiety through a linker reported to be useful for the treatment of cancer. Read More
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. Read More
Purdue Research Foundation has identified conjugates targeting asialoglycoprotein receptors (ASGR) that are comprised of a therapeutic or imaging agent. They are reported to be potentially useful for the diagnosis and/or treatment of nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH). Read More
Adlai Nortye Biopharma Co. Ltd. and Adlai Nortye Pte Ltd. have divulged cyclin-dependent kinase (CDK) inhibitors reported to be useful for the treatment of cancer, inflammatory disorders and autoimmune diseases. Read More
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. Read More
Suzhou Genhouse Bio Co. Ltd. has described histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of cancer, asthma, Alzheimer’s disease, diabetes, amyotrophic lateral sclerosis, multiple sclerosis, pulmonary fibrosis and psoriasis, among others. Read More
