https://www.fda.gov/media/151969/download
Adeno-associated Virus (AAV) and AAV Vectors
AAV is a small DNA virus (20 - 25 nm diameter, ~ 5 kb genome)
o Widespread in humans and animals
o More than 100 serotypes, with tropisms for different cell types
o Only replicates in the presence of a helper virus (e.g., adenovirus, HSV-1,
•
HHV-6, EBV)
o No known disease association
AAV vectors
o Do not replicate
o Are engineered to express a therapeutic gene
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Summary
• Serious and life-threatening systemic toxicities have been reported as more patients receive AAV vectors, and as vector doses increase.
• There are many questions about these toxicities,including how to: o Understand mechanisms
o Mitigate risks to participants in clinical trials
• FDA is seeking the committee’s insights into AAV vector toxicities in the context of:
o AAV vector design and quality
o Preclinical and clinical study designs
o Long-term monitoring of individuals receiving these products
