Accelerating Research & Development for Advanced Therapies

Project Information

ARDAT

Grant agreement ID: 945473

DOI

10.3030/945473

EC signature date 11 November 2020

Start date 1 November 2020

End date 31 October 2025

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Total cost

€ 25 500 169,75

EU contribution

€ 11 772 999,75

Coordinated by

THE UNIVERSITY OF SHEFFIELD
United Kingdom

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Deliverables

Documents, reports (2)

Ethics Report - 4th Periodic Reporting

Ethics report produced by the Ethics Advisor every reporting period as outlined in D1.10.

Publication of current protocols with the associated burden/risk/cost

Websites, patent fillings, videos etc. (1)

Workshop 2 with stakeholders

Organisation of Workshop 2 with stakeholders

Publications

Peer reviewed articles (13)

Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B

Author(s): Ulrike M. Reiss, Andrew M. Davidoff, Edward G.D. Tuddenham, Pratima Chowdary, Jenny McIntosh, Vincent Muczynski, Malo Journou, Giulia Simini, Lydia Ireland, Saira Mohamed, Anne Riddell, Arnulfo J. Pie, Andrew Hall, Alberto Quaglia, Sarah Mangles, Johnny Mahlangu, Kristina Haley, Michael Recht, Yu-Min Shen, Kathleen G. Halka, Gail Fortner, Christopher L. Morton, Zhengming Gu, Randall T. Hayden, Ell
Published in: New England Journal of Medicine, Issue 392, 2025, Page(s) 2226-2234, ISSN 0028-4793
Publisher: Massachusetts Medical Society
DOI: 10.1056/nejmoa2414783

The IgG-degrading enzyme, Imlifidase, restores the therapeutic activity of FVIII in inhibitor-positive hemophilia A mice

Author(s): Bou-Jaoudeh, Melissa; Delignat, Sandrine; Daventure, Victoria; Astermark, Jan; Lévesque, Hervé; Dimitrov, Jordan, D; Deligne, Claire; Proulle, Valérie; Lacroix-Desmazes, Sébastien
Published in: Haematologica, Issue Volume 108, Number 5, 2023, ISSN 1592-8721
Publisher: Ferrata Storti Foundation
DOI: 10.3324/haematol.2022.281895

Evading and overcoming AAV neutralization in gene therapy

Author(s): Joseph Earley, Elena Piletsku, Giuseppe Ronzetti, sergey Piletsky
Published in: Trends in Biotechnology, Issue Vol 41, June 2023, 2023, Page(s) 836 - 845, ISSN 0167-9430
Publisher: Elsevier
DOI: 10.1016/j.tibtech.2022.11.006

Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47

Author(s): Jessica P Wiseman, Joseph M Scarrott, João Alves-Cruzeiro, Afshin Saffari, Cedric Böger, Evangelia Karyka, Emily Dawes, Alexandra K Davies , Paolo M Marchi, Emily Graves, Fiona Fernandes, Zih-Liang Yang, Ian Coldicott, Jennifer Hirst, Christopher P Webster, J Robin Highley, Neil Hackett, Adrienn Angyal, Thushan de Silva, Adrian Higginbottom, Pamela J Shaw, Laura Ferraiuolo, Darius Ebrahimi-Fakha
Published in: EMBO Molecular Medicine, Issue Volume 16, Number 11, 2024, Page(s) 2882 – 2917, ISSN 1757-4684
Publisher: Nature Publishing Group UK on behalf of EMBO Press
DOI: 10.1038/s44321-024-00148-5

Current global regulatory landscape for biodistribution and shedding assessment of rAAV gene therapies and recommendations of the IMI ARDAT consortium on future directions

Author(s): Natalie Anne Schmidt, Jon Giblin, Timothy K MacLachlan, Shera Dandapat, Gabor Veres, Tatiana Anna Reimer, Martin Schulz, Eva Hatzmann Andeleeb Dahy Gregory LaRosa, Mimoun Azzouz and Christopher J Mann
Published in: Cell and Gene Therapy Insights, Issue 20597800, 2022, Page(s) 377-394, ISSN 2059-7800
Publisher: Bioinsights Publishing Ltd
DOI: 10.18609/cgti.2022.056

Half‐Life Extension of the IgG‐Degrading Enzyme (IdeS) Using Fc‐Fusion Technology

Author(s): Victoria Daventure, Melissa Bou‐Jaoudeh, Emna Hannachi, Alejandra Reyes‐Ruiz, Amélia Trecco, Sandrine Delignat, Sébastien Lacroix‐Desmazes, Claire Deligne
Published in: European Journal of Immunology, Issue 55, 2025, ISSN 0014-2980
Publisher: John Wiley & Sons Ltd.
DOI: 10.1002/eji.202451264

Polyfunctional T cells and unique cytokine clusters imprint the anti rAAV2/rAAV9 vector immune response

Author(s): Stephan J. Holtkamp, Florian R. Lagoda, Adam Lister, Pradeep Harish, Ulrike Kleymann, Theresa Pesch, Chai Fen Soon, Munir Pirmohamed, Dean Naisbitt, Mark Trautwein
Published in: Frontiers in Immunology, Issue 15, 2024, ISSN 1664-3224
Publisher: Frontiers in Immunology
DOI: 10.3389/fimmu.2024.1450524

AAV vectors trigger DNA damage responses and STING-dependent inflammation in human CNS cells

Author(s): Helena Costa-Verdera, Vasco Meneghini, Zachary Fitzpatrick, Monah Abou Alezz, Emily Fabyanic, Xin Huang, Yulia Dzhashiashvili, Avantika Ahiya, Elisabeth Mangiameli, Erika Valeri, Giovanni Crivicich, Ivan Cuccovillo, Roberta Caccia, Berangere Bertin, Giuseppe Ronzitti, Esteban Engel, Ivan Merelli, Federico Mingozzi, Angela Gritti, Klaudia Kuranda, Anna Kajaste-Rudnitski
Published in: Nature Communications, 2025, Page(s) 20, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.21203/rs.3.rs-4171795/v1

A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders

Author(s): Besarte Vrellaku, Ilda Sethw Hassan, Rebecca Howitt, Christopher P. Webster, Eli Harriss, Fraser McBlane, Corinne Betts, Jorge Schettini, Mattia Lion, John E. Mindur, Michael Duerr, Pamela J. Shaw, Janine Kirby, Mimoun Azzouz, Laurent Servais
Published in: Molecular Therapy, Issue Volume 32, Issue 10, 2024, Page(s) 3220-3259, ISSN 1525-0024
Publisher: Cell Press
DOI: 10.1016/j.ymthe.2024.07.016

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

Author(s): David-Alexandre Gross, Novela Todesco, Christiean Leboorgne, Guiseppe Ronzitti
Published in: Frontiers in Immunology, Issue 07 April 2022, 2022, ISSN 1664-3224
Publisher: Lausanne: Frontiers Media SA
DOI: 10.3389/fimmu.2022.857276

Imlifidase, a new option to optimize the management of patients with hemophilia A on emicizumab

Author(s): Melissa Bou-Jaoudeh, Angelina Mimoun, Sandrine Delignat, Peter J. Lenting, Valérie Proulle, Sébastien Lacroix-Desmazes
Published in: Journal of Thrombosis and Haemostasis, Issue Volume 21, Issue 10, 2023, ISSN 1538-7836
Publisher: Oxford Elsevier
DOI: 10.1016/j.jtha.2023.06.038

A microvascularized in vitro liver model for disease modeling and drug discovery

Author(s): Flavio Bonanini, Roelof Dinkelberg, Manuel Caro Torregrosa, Nienke Kortekaas, Tessa M. S. Hagens, Stéphane Treillard, Dorota Kurek, Vincent van Duinen, Paul Vulto, Kristin Bircsak
Published in: Biofabrication, Issue Volume 17, Number 1, 2024, ISSN 1758-5090
Publisher: IOP Publishing Ltd
DOI: 10.1088/1758-5090/ad818a

Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver

Author(s): Louisa Jauze, Mallaury Vie, Quentin Miagoux, Lucille Rossiaud, Patrice Vidal, Valle Montalvo-Romeral, Hanadi Saliba, Margot Jarrige, Helene Polveche, Justine Nozi, Pierre-Romain Le Brun, Luca Bocchialini, Amandine Francois, Jérémie Cosette, Jérémy Rouillon, Fanny Collaud, Fanny Bordier, Emilie Bertil-Froidevaux, Christophe Georger, Laetitia van Wittenberghe, Adeline Miranda, Nathalie F. Daniel
Published in: JCI Insight, Issue 9, 2024, ISSN 2379-3708
Publisher: JCI Insight
DOI: 10.1172/jci.insight.172614