Notable advances have been reported in therapies for neurological and orphan diseases. Biogen shared encouraging early Phase 1 data for salanersen, an antisense oligonucleotide aimed at spinal muscular atrophy, potentially benefiting patients insufficiently helped by existing treatments. An AAV RNA interference approach targeting ataxin-2 significantly extended survival in a mouse model of TDP-43 proteinopathy, relevant to ALS and frontotemporal dementia. Additionally, promising preclinical results were disclosed for a WEE1 inhibitor targeting HPV-positive head and neck cancer. These developments underscore an expanded therapeutic pipeline addressing unmet needs in neurodegeneration and oncology.