FDA Approves Eight AAV Gene Therapies Under BLAs
Between 2017 and 2025, the US Food and Drug Administration approved eight adeno-associated virus-based in vivo gene therapy products under biologics license applications, Nature reported. The regulatory agency also received over 300 new investigational new drug submissions for gene delivery using AAV by the end of 2025. These figures mark a significant period of clinical progression for genetic medicines, prompting public and FDA data analyses to guide future submissions and speed up patient access.
Despite this clinical momentum, the field of rare diseases still has severe unmet needs. Over 10,000 identified rare conditions affect millions of people globally, yet only 5% of rare diseases have FDA-approved therapies, according to policy statements published by the American Society of Gene and Cell Therapy. The ASGCT paper was authored by Diane Berry of Sarepta Therapeutics, Kate Donigan of Sarepta, Lisa Kahlman of Ultragenyx Pharmaceutical, James Long of DK Pierce, Christina Markus of King & Spalding, and Caitlin K. McCombs of the ASGCT.
Adapting regulatory frameworks for gene therapy trials
Traditional regulatory paradigms often fail to match the distinctive nature of gene therapies for rare and ultrarare diseases. Clinical trial designs, endpoint selections, and manufacturing processes for these advanced modalities demand non-traditional approaches. The rarity of the conditions, alongside their heterogeneous and progressive nature, requires modern, nimble strategies to evaluate safety and efficacy. Regulatory frameworks must adapt to accommodate platform technologies and iterative manufacturing evaluations.
Precision genetic medicine addresses the root causes of serious genetic disorders, offering pathways to alter disease trajectories. However, the journey from scientific breakthrough to the patient bedside faces unique obstacles in oversight. Flexible manufacturing processes and a holistic evidence approach are critical to aligning regulatory requirements with the rapid pace of science.
Frequently Asked Questions About AAV Gene Therapies
What is an adeno-associated virus vector in gene therapy?
Between 2017 and 2025, the FDA approved eight such in vivo products under biologics license applications.
How many investigational new drug submissions has the FDA received for AAV delivery?
The FDA received over 300 new investigational new drug submissions utilizing AAV gene delivery by the end of 2025. These submissions aim to advance clinical candidates through regulatory pipelines.
What proportion of rare diseases currently have FDA-approved treatments?
Only 5% of the more than 10,000 identified rare diseases have an FDA-approved therapy available, according to the ASGCT policy analysis. This creates an urgent unmet need for optimized regulatory pathways.
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