Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain
the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in
Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles
and JavaScript.
4BIO Capital LLP, London, UK; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
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.
Prices may be subject to local taxes which are calculated during checkout
Nature Reviews Drug Discovery20, 173-174 (2021)
doi: https://doi.org/10.1038/d41573-021-00017-7
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.
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.