By mid-2026, the industry has officially entered the era of "In Vivo Liver Editing." This year, the focus is on Clinical Phase 1/2 Gene Therapy trials (such as BioMarin’s BMN 307 and various AAV-mediated platforms) that aim to permanently restore PAH enzyme activity in the liver. This innovation is a primary driver for the market, as patients are increasingly demanding a "One-and-Done"...
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