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. Read More

Dark genome miner Enara Bio Ltd. has closed a $32.5 million series B that will see the lead program targeting the first of a novel class of cancer antigens it has discovered through to the clinic. Enara calls these cancer antigens “dark antigens” (the name is trademarked). It says they can be found in solid tumors irrespective of the immune phenotype, and are often expressed at high prevalence across multiple different tumors. Read More

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. Read More

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. Read More

At this week’s American Society for Radiation Oncology meeting, scientists from The University of Texas MD Anderson Cancer Center reported the discovery and preclinical evaluation of CD47-LLO, a novel microbial-inspired antibody-drug conjugate (ADC) for the treatment of cancer. Read More

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. Read More

Deliver Therapeutics Inc. has disclosed protein kinase inhibitors reported to be useful for leukemia. Read More

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. Read More

Scientists at CeMM-Forschungsinstitut für Molekulare Medizin GmbH and Max Planck Society for the Advancement of Science EV have synthesized proteolysis targeting chimera (PROTAC) compounds comprising a DDB1- and CUL4-associated factor 11 (DCAF11) binding moiety covalently linked to a different protein targeting moiety through a linker. Read More

Nutshell Biotech (Shanghai) Co. Ltd. has identified nitrogen-containing macrocyclic compounds acting as DNA polymerase θ (POLθ) inhibitors reported to be useful for the treatment of cancer. Read More

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. Read More

RTI International has divulged vancomycin derivatives with siderophore modification reported to be useful for the treatment of gram-negative and gram-positive bacterial infections. Read More

Foundation for Neglected Disease Research has described flavonoid compounds reported to be useful for the treatment of viral infections. Read More

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. Read More

It has been previously demonstrated that intranasal treatment with COG-201, an AAV9-shRNA designed to target the down-regulation of the 5-HT2A receptor, significantly decreased anxiety and improved memory in mice and rats. Read More