2025 delivered notable progress for in‑vivo gene therapies as developers reported improved delivery methods and encouraging preclinical and early clinical data, signaling momentum for one‑time genetic treatments. Multiple groups documented advances in tissue targeting and payload design that improved on‑target editing rates. At the same time, the field continues to debate which gene‑editing modality will prevail. Researchers and industry leaders described a crowded landscape of CRISPR variants, base editors, prime editors and novel nucleases, with no single 'final' technology yet dominant. Investors and developers will track delivery platforms and safety readouts closely; delivery remains the principal bottleneck for in‑vivo approaches and will determine which editing tools can be translated safely to patients. For context, 'in‑vivo' refers to gene therapies delivered directly into patients rather than via ex‑vivo manipulated cells.
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