Gene therapy using adeno-associated virus (AAV) as a vector has emerged as a novel therapeutic modality that has the potential to lead to substantial disease modification in many monogenic disorders, or perhaps even cures. Given the interest in the approach, which has been boosted by the recent approval of two AAV-based gene therapies by the US FDA, we have conducted a systematic review of the landscape of clinical trials of AAV-based gene therapies (see Supplementary Fig. 1 and Supplementary Table 1 for details). Here, we highlight the key trends and discuss the implications.
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Acknowledgements
The authors thank R. Kerr (University of Oxford), A. Bersenev (Yale University) and D. M. Kullmann (University College London) for their input into the study design and discussion of the dataset.
Competing Interests
D.A.K. is a non-executive director of Redpin Therapeutics, which develops AAV gene therapies. O.P.S. is a non-executive director of SparingVision SA, which develops AAV gene therapies. J.C.M. vdL is a consultant to 4BIO Capital. The other authors declare no competing interests.
