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Gene therapy is entering its most transformative era yet, with AI-designed proteins, precise CRISPR editing and approved treatments for retinal disorders, spinal muscular atrophy and blood cancers proving the science works. Viral and non-viral delivery systems keep improving in safety and efficiency, expanding the range of treatable diseases fast. Market-driven biotech innovation is accelerating cures at a pace no government program could match.
Gene therapy's promise is being strangled by broken economics and political neglect. Per-patient costs can run into seven figures, and companies like Prime Medicine are already halting work on successful treatments because the business model doesn't hold up. Only a publicly funded health system treating gene therapy like dialysis or transplants — a collective service, not a market commodity — can turn lab breakthroughs into medicine for everyone.