Clinical gene technology in Australia: building on solid foundations.

"Competing interests John Rasko has served as Chair on the Therapeutic Goods Administration Advisory Committee on Biologicals and the National Health and Medical Research Council Mitochondrial Donation Expert Working Committee. As Head of Department, Cell and Molecular Therapies, Royal Prince Alfred Hospital (RPAH), and as Head of the Gene and Stem Cell Program at the Centenary Institute, he is responsible for undertaking investigator and pharmaceutical companies‐sponsored clinical trials of immunotherapies, cellular therapies and gene therapies at RPAH and basic research at the Centenary Institute, often in collaboration with biotechnology and infrastructure companies. He has also had Material Transfer Agreements, consultancy or honoraria with a number of companies, including Gilead, Novartis, Bluebird bio, Spark Therapeutics, Cynata, Pfizer, Atara Bio, Bayer and CRISPR Therapeutics. Any patents are held by the Sydney Local Health District, the Centenary Institute or the University of Sydney as his primary affiliations. He has extensive research and clinical interests in gene technology, especially in haemophilia gene therapy and CAR‐T cells. Gabrielle O’Sullivan has served on the Gene Technology Technical Advisory Committee since 2011, including as cross‐member of the Gene Technology Ethics and Community Consultative Committee from 2015 to the present. Joshua Philips is employed by the Office of the Gene Technology Regulator."

"John Rasko acknowledges grant funding from the National Health and Medical Research Council (Investigator Grant 1177305) “Driving clinical cell and gene therapy in Australia” and from Therapeutic Innovation Australia. These funding sources support John Rasko in developing the fields of cell and gene therapy in Australia, and had no specific role in this submitted work. We thank Michael Dornbusch and Heidi Mitchell of the Office of the Gene Technology Regulator for providing advice and guiding the development of this article. We also thank Charles Bailey and Bijay Dhungel of the Gene and Stem Cell Therapy Program at Centenary Institute for providing advice and assistance with the illustrations. The views expressed in the article are those of the authors alone and do not represent an official view of the Australian Government, the Gene Technology Regulator, New South Wales Health, the Sydney Local Health District or the Royal Prince Alfred Hospital."