A new gene‑therapy study reported functional restoration of SynGAP1 and rescue of epilepsy and associated behavioral deficits in preclinical models. SynGAP1‑related disorders cause intellectual disability and intractable epilepsy; the therapy delivered gene replacement that normalized molecular pathways and improved seizure outcomes. The results, published in translational neuroscience outlets, mark another step toward clinical strategies for neurodevelopmental disorders caused by single‑gene loss‑of‑function. Sponsors and rare‑disease investors will watch safety, dosing, and long‑term efficacy data as programs advance toward first‑in‑human trials.