Industry reviews label 2025 a breakthrough year for in‑vivo gene therapies, citing multiple preclinical and clinical advances that improved delivery, specificity and efficacy. In that landscape, a gene‑replacement approach restored SynGAP1 function and rescued epilepsy‑related phenotypes in preclinical models, offering a translational example of how genetic restoration can reverse neurological disease features. Together these items point to accelerating translational pipelines for monogenic and complex disorders, with attention on delivery technology, safety readouts, and scalable manufacturing as the next hurdles for clinical translation.