Story perspectives

Gene Therapy Faces Setbacks, Yet Promising Alternatives Emerge

10/24/2025

Summary

Story summary

AAV gene therapy is facing significant challenges after a decade of promise, including high costs and safety concerns.
Biogen and Pfizer have halted AAV programs, citing economic and clinical hurdles.
Despite setbacks, gene therapy for neovascular age-related macular degeneration (nAMD) shows potential with trials reducing treatment burden.
New alternatives like lipid nanoparticles and non-viral delivery systems are emerging as viable options, while manufacturing and regulation need improvement.

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Story summary

AAV gene therapy is facing significant challenges after a decade of promise, including high costs and safety concerns.
Biogen and Pfizer have halted AAV programs, citing economic and clinical hurdles.
Despite setbacks, gene therapy for neovascular age-related macular degeneration (nAMD) shows potential with trials reducing treatment burden.
New alternatives like lipid nanoparticles and non-viral delivery systems are emerging as viable options, while manufacturing and regulation need improvement.