The discovery of a complex formed by two types of ion channels in neurons has allowed researchers from Heidelberg University to develop an inhibitor that stopped motor neuron degeneration in amyotrophic lateral sclerosis (ALS) in mouse models and human brain organoids.

Bruton tyrosine kinase (BTK) enzyme inhibitors used to treat B-cell cancers, including chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma, also produce resistance by causing mutations in the protein. Now, a study on the BTK degrader NX-2127 showed the compound could be effective in eliminating BTK regardless of its mutations.

Spyglass Ophthalmics Inc. is seeking patent protection for intraocular drug delivery devices and methods of delivering therapeutic agents to the eye. The methods include implanting an intraocular implant into the eye and adjacent to a fluid-permeable membrane of the patient’s eye.

The Foundation for the National Institutes of Health (FNIH) has announced the online publication of the first playbook designed to help accelerate the development of adeno-associated virus (AAV) gene therapies for rare diseases.

The European Chemicals Agency (ECHA) has contracted a consortium to conduct scientific studies on the reliability and relevance of new approach methodologies (NAMs) as alternatives to animal testing and to promote the use of such methods in the future.

CAR T cells could be repurposed to target senescent cells and delay the effect of aging. A study by scientists at Cold Spring Harbor Laboratory showed how to design them and demonstrated the advantages of this therapy in mice. “We only gave one dose, and we could have benefits [for] really long periods of time,” lead author Corina Amor told BioWorld.

Researchers from the Institute for Bioengineering of Catalonia (IBEC) and collaborators have successfully treated bladder tumors in mice using urease-powered nanobots. The testing consisted of the administration of 18F-nanorobots to tumor-bearing mice divided into four groups depending on tumor volumes using a group of non-tumor-bearing mice as control. Importantly, the therapy remained in the bladder with only a very small proportion of radioactivity seen in other organs. The results were published in Nature Nanotechnology on Jan. 15, 2024.

Researchers from Broad Institute and Harvard University presented the discovery of all-in-one virus-like particles (VLPs) designed to deliver prime editor (PE) ribonucleoprotein (RNP) complexes into mammalian cells.