Researchers highlighted the potential of targeted in vivo gene‑editing interventions to address life‑threatening congenital and early‑onset diseases—presentations at ASGCT emphasized preclinical and translational advances that may enable fetal and neonatal interventions before irreversible damage occurs. Industry moves to secure platform access followed: Toolgen and Geneditbio signed a strategic cross‑license to jointly advance next‑generation in vivo genome‑editing therapeutics, combining intellectual property and delivery know‑how. The agreement aims to accelerate IND‑enabling programs and broaden the scope of editable targets. These scientific advances and IP deals together demonstrate momentum toward clinically deployable in vivo editing, but highlight remaining hurdles in targeted delivery, regulatory clarity and long‑term safety surveillance.