Novel therapeutics targeting respiratory and neurodegenerative diseases have gained momentum. Insmed’s first DPP1 inhibitor brensocatib offers a new option for bronchiectasis patients. Meanwhile, a mitochondria-targeted receptor developed by Inserm boosts mitochondrial activity and reverses cognitive impairment in mouse models of Alzheimer’s and frontotemporal dementia. Concurrently, RNA-based therapies evolve, including brain-penetrant RBM39 degraders entering clinical trials and innovative organotypic brain slice culture models that enable live-cell imaging, supporting better disease understanding and treatment development.