Technological advances are accelerating gene and RNA-based therapies. Researchers demonstrated a near-infrared light activatable CRISPR system enabling precise spatiotemporal genome editing. Another study highlighted that slowing intracellular transport enhances efficacy of antisense oligonucleotides (ASOs) by prolonging endosomal residence time. The US FDA continues to push the transition from mandatory animal testing to New Approach Methodologies (NAMs), favoring computational and organoid models, facilitating innovation in gene medicine development and regulatory policy.