Nature Biotechnology published work demonstrating in vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles, marking a technical advance toward non-conditioned in vivo editing of blood-lineage cells. The method shows potential to simplify delivery and avoid ex vivo manufacturing bottlenecks. Separately, Encoded Therapeutics reported Phase 1/2 Dravet syndrome gene therapy data linking ETX101 to a 78% reduction in seizures, positioning the company to start a pivotal trial next year. Encoded’s clinical progress highlights the translational pathway from novel delivery platforms and preclinical editing technologies to actionable programs in severe pediatric neurological disease. Taken together, the two reports indicate concurrent progress in both delivery science and clinical-stage gene therapies, suggesting an accelerating pipeline of in vivo and ex vivo modalities entering pivotal development.