Immune checkpoint inhibitors targeting the PD-1/PD-L1 pathway are widely used in cancer immunotherapy, but poor tissue distribution or immune-related adverse events often limit their application.

The signaling of TNF receptor (TNFR) superfamily member 4 (OX40) and its ligand (OX40L) plays a crucial role in the development of immunological and inflammatory disorders due to triggering a subset of T-cell responses.

As in other muco-obstructive diseases, the airways in cystic fibrosis (CF) are characterized by goblet cell and glandular hyperplasia, with overproduction of mucins MUC5 and MUC5AC, resulting in viscous mucus, respiratory blockade and recurrent infections and inflammation.

Gigagen Inc., a subsidiary of Grifols SA, has been awarded a contract by the U.S. Biomedical Advanced Research and Development Authority (BARDA) to develop a recombinant polyclonal antibody therapy for botulinum neurotoxins and a second biothreat of interest yet to be determined.

Integrated Biosciences Inc. has closed a $17.2 million seed financing to support its work combining synthetic biology and artificial intelligence (AI)-driven small-molecule drug discovery to produce next-generation therapeutics targeting age-related diseases.

The increasing knowledge on how protein tau is organized in live cells has shown that the protein forms nanometer-sized hotspots which are different from tau microtubules. These hotspots, essential for aggregation, include (306)VQIVYK(311) and (275)VQIINK(280) aggregation-promoting hotspots, the first found in all tau isoforms and the latter included mainly in 4R isoforms.

Researchers in the U.K. have succeeded in reverse engineering the defective cryptic splicing that drives amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) to enable precisely targeted delivery of transgenes and therapeutic protein expression in diseased neurons. The technique is compatible with conventional adeno-associated viral vectors that are approved for gene therapy, and can readily be adapted for different transgenes. ALS, FTD and other neurogenerative diseases are underpinned by loss of function of the RNA-binding protein TDP-43 (transactive response DNA-binding protein 43), that normally functions as a key regulator of splicing, protecting the transcriptome from toxic cryptic exons.

Immunotherapy company Cartherics Pty Ltd. raised AU$15 million (US$10.3 million) in an oversubscribed series B round that will support the first clinical trial for lead chimeric antigen receptor natural killer therapy CTH-401 for ovarian cancer, and to expand its pipeline to include other diseases. Cartherics CEO Alan Trounson told BioWorld that the funds raised will take Cartherics through to mid-2026, and the phase I Australian trial in ovarian cancer will begin in the fourth quarter of 2025.