Commentary & Perspectives

Bringing New Ideas to AAV Gene Therapy

As safety concerns and commercial doubts threaten the AAV gene therapy field, new technologies may offer a “well-rounded” solution.

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In 2025, the AAV gene therapy field has faced significant challenges, including severe toxicity leading to fatalities in clinical trials and a dramatic decline in venture capital investments by over 80%. Major companies like Pfizer and Biogen are exiting the space, raising questions about the future of AAV therapy. Key issues include commercial viability, high development costs, and technical challenges related to gene delivery and expression levels. Despite setbacks, ongoing academic and industry efforts aim to refine AAV capsids and utilize circular RNA to enhance therapeutic effectiveness and safety, paving the way for a potential resurgence in AAV gene therapy.

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