Accelerating Research & Development for Advanced Therapies

Informazioni relative al progetto

ARDAT

ID dell’accordo di sovvenzione: 945473

DOI

10.3030/945473

Progetto chiuso

Data della firma CE 11 Novembre 2020

Data di avvio 1 Novembre 2020

Data di completamento 31 Luglio 2026

Finanziato da

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

Costo totale

€ 25 500 169,75

Contributo UE

€ 11 772 999,75

Coordinato da

THE UNIVERSITY OF SHEFFIELD
United Kingdom

CORDIS fornisce collegamenti ai risultati finali pubblici e alle pubblicazioni dei progetti ORIZZONTE.

I link ai risultati e alle pubblicazioni dei progetti del 7° PQ, così come i link ad alcuni tipi di risultati specifici come dataset e software, sono recuperati dinamicamente da .OpenAIRE .

Risultati finali

Documents, reports (3)

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
Whitepaper 2 (immunosuppression protocols)

Whitepaper 2 (immunosuppression protocols)

Websites, patent fillings, videos etc. (1)

Workshop 2 with stakeholders

Organisation of Workshop 2 with stakeholders

Pubblicazioni

Peer reviewed articles (13)

Current regulatory requirements for assessment of immunogenicity for gene therapy medicinal products

Autori: Christopher J. Mann, Jon Giblin, Manuela Braun, Felicitas Schmid, Maria Rathmann Sørensen, Paolo Caferra, Anett Hudák, Tamás Letoha, Núria Coderch, Timothy P. Hickling, Mimoun Azzouz
Pubblicato in: Cell Reports Medicine, 2025, Pagina/e 102422, ISSN 2666-3791
Editore: Elsevier BV
DOI: 10.1016/j.xcrm.2025.102422

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

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

Evading and overcoming AAV neutralization in gene therapy

Autori: Joseph Earley, Elena Piletsku, Giuseppe Ronzetti, sergey Piletsky
Pubblicato in: Trends in Biotechnology, Numero Vol 41, June 2023, 2023, Pagina/e 836 - 845, ISSN 0167-9430
Editore: Elsevier
DOI: 10.1016/j.tibtech.2022.11.006

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

Autori: 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
Pubblicato in: EMBO Molecular Medicine, Numero Volume 16, Number 11, 2024, Pagina/e 2882 – 2917, ISSN 1757-4684
Editore: 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

Autori: 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
Pubblicato in: Cell and Gene Therapy Insights, Numero 20597800, 2022, Pagina/e 377-394, ISSN 2059-7800
Editore: Bioinsights Publishing Ltd
DOI: 10.18609/cgti.2022.056

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

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

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

Autori: Stephan J. Holtkamp, Florian R. Lagoda, Adam Lister, Pradeep Harish, Ulrike Kleymann, Theresa Pesch, Chai Fen Soon, Munir Pirmohamed, Dean Naisbitt, Mark Trautwein
Pubblicato in: Frontiers in Immunology, Numero 15, 2024, ISSN 1664-3224
Editore: Frontiers in Immunology
DOI: 10.3389/fimmu.2024.1450524

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

Autori: 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
Pubblicato in: Molecular Therapy, Numero Volume 32, Numero 10, 2024, Pagina/e 3220-3259, ISSN 1525-0024
Editore: Cell Press
DOI: 10.1016/j.ymthe.2024.07.016

Pre-existing humoral immunity and complement pathway contribute to immunogenicity of adeno-associated virus (AAV) vector in human blood

Autori: Corinne J. Smith, Nikki Ross, Ali Kamal, Kevin Y. Kim, Elizabeth Kropf, Pascal Deschatelets, Cedric Francois, William J. Quinn, Inderpal Singh, Anna Majowicz, Federico Mingozzi, Klaudia Kuranda
Pubblicato in: Frontiers in Immunology, Numero 13, 2023, ISSN 1664-3224
Editore: Frontiers in Immunology
DOI: 10.3389/fimmu.2022.999021

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

Autori: David-Alexandre Gross, Novela Todesco, Christiean Leboorgne, Guiseppe Ronzitti
Pubblicato in: Frontiers in Immunology, Numero 07 April 2022, 2022, ISSN 1664-3224
Editore: 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

Autori: Melissa Bou-Jaoudeh, Angelina Mimoun, Sandrine Delignat, Peter J. Lenting, Valérie Proulle, Sébastien Lacroix-Desmazes
Pubblicato in: Journal of Thrombosis and Haemostasis, Numero Volume 21, Numero 10, 2023, ISSN 1538-7836
Editore: Oxford Elsevier
DOI: 10.1016/j.jtha.2023.06.038

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

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

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

Autori: 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
Pubblicato in: JCI Insight, Numero 9, 2024, ISSN 2379-3708
Editore: JCI Insight
DOI: 10.1172/jci.insight.172614