Research
A comprehensive analysis of CBER experience with AAV gene therapy IND submissions
Nature · By Sojin Bing ·
The Brief
- An analysis of Investigational New Drug (IND) submissions to the US FDA from 1995 to 2025 examines CBER's experience with AAV gene therapy.
- The analysis reports rapid growth in adeno-associated virus (AAV) development.
- The study was published online in Nature Biotechnology on 12 November 2026.
Why it matters
The analysis offers a data-based view of how AAV gene therapy programmes have moved through FDA IND submissions over three decades, which is useful context for researchers planning or assessing gene therapy trials.
Who’s affected
- researchers and developers of AAV gene therapies
- people following FDA regulation of gene therapies
Nature Biotechnology, Published online: 12 November 2026; doi:10.1038/s41587-026-03378-9 An analysis of Investigational New Drug (IND) submissions to the US Food and Drug Administration (FDA) from 1995 to 2025 reveals a rapid growth in adeno-associated virus (AAV) development…
Timeline
Analysis published online in Nature Biotechnology
nature.com
A comprehensive analysis of CBER experience with AAV gene therapy IND submissions