DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID)

Deliverables

Efficacy of Artemis clinical batch and ODD status
Implementation of CliniMACS Prodigy in clinical trial
Correct OS in mouse model and patient cells
A set of clinically applicable vectors that can correct RAG1 and RAG2 deficiency in mouse models and human cells
Gene editing protocol for one disease entity
Website for SCIDNET
Minutes of 1st General Assembly meeting
Minutes of first SAB
Minutes of third SAB
Minutes of second SAB
Project kick-off meeting minutes

Initiation of clinical trial
Setting up of SCIDNET project website

Publications

Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

Author(s): Valentina Capo, Maria Carmina Castiello, Elena Fontana, Sara Penna, Marita Bosticardo, Elena Draghici, Luigi P. Poliani, Lucia Sergi Sergi, Rosita Rigoni, Barbara Cassani, Monica Zanussi, Paola Carrera, Paolo Uva, Kerry Dobbs, Nicolò Sacchetti, Luigi D. Notarangelo, Niek P. van Til, Gerard Wagemaker, Anna Villa
Published in: Journal of Allergy and Clinical Immunology, Issue 142/3, 2018, Page(s) 928-941.e8, ISSN 0091-6749
Publisher: Mosby Inc.
DOI: 10.1016/j.jaci.2017.11.015

Generation of adult human T-cell progenitors for immunotherapeutic applications

Author(s): Laura Simons, Kuiying Ma, Corinne de Chappedelaine, Ranjita Devi Moiranghtem, Elodie Elkaim, Juliette Olivré, Sandrine Susini, Kevin Appourchaux, Christian Reimann, Hanem Sadek, Olivier Pellé, Nicolas Cagnard, Elisa Magrin, Chantal Lagresle-Peyrou, Tom Taghon, Antonio Rausell, Marina Cavazzana, Isabelle André-Schmutz
Published in: Journal of Allergy and Clinical Immunology, Issue 141/4, 2018, Page(s) 1491-1494.e4, ISSN 0091-6749
Publisher: Mosby Inc.
DOI: 10.1016/j.jaci.2017.10.034

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Author(s): Annalisa Lattanzi, Vasco Meneghini, Giulia Pavani, Fatima Amor, Sophie Ramadier, Tristan Felix, Chiara Antoniani, Cecile Masson, Olivier Alibeu, Ciaran Lee, Matthew H. Porteus, Gang Bao, Mario Amendola, Fulvio Mavilio, Annarita Miccio
Published in: Molecular Therapy, Issue 27/1, 2019, Page(s) 137-150, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2018.10.008

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

Author(s): Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio
Published in: Molecular Therapy - Methods & Clinical Development, Issue 9, 2018, Page(s) 257-269, ISSN 2329-0501
Publisher: Elsevier
DOI: 10.1016/j.omtm.2018.03.002

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

Author(s): Frank J. T. Staal, Alessandro Aiuti, Marina Cavazzana
Published in: Frontiers in Pediatrics, Issue 7, 2019, ISSN 2296-2360
Publisher: Frontiers in Pediatrics
DOI: 10.3389/fped.2019.00443

Concise Review: BOOSTING T Cell Reconstitution Following Allogeneic Transplantation—Current Concepts and Future Perspectives

Author(s): Laura Simons, Marina Cavazzana, Isabelle André
Published in: STEM CELLS Translational Medicine, 2019, ISSN 2157-6564
Publisher: AlphaMed Press
DOI: 10.1002/sctm.18-0248

Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency

Author(s): Ornellie Bernadin, Fouzia Amirache, Anais Girard-Gagnepain, Ranjita Devi Moirangthem, Camille Lévy, Kuiying Ma, Caroline Costa, Didier Nègre, Christian Reimann, David Fenard, Agata Cieslak, Vahid Asnafi, Hanem Sadek, Rana Mhaidly, Marina Cavazzana, Chantal Lagresle-Peyrou, François-Loïc Cosset, Isabelle André, Els Verhoeyen
Published in: Blood Advances, Issue 3/3, 2019, Page(s) 461-475, ISSN 2473-9529
Publisher: Blood
DOI: 10.1182/bloodadvances.2018027508

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

Author(s): Sabine Charrier, Chantal Lagresle-Peyrou, Valentina Poletti, Michael Rothe, Grégory Cédrone, Bernard Gjata, Fulvio Mavilio, Alain Fischer, Axel Schambach, Jean-Pierre de Villartay, Marina Cavazzana, Salima Hacein-Bey-Abina, Anne Galy
Published in: Molecular Therapy - Methods & Clinical Development, Issue 15, 2019, Page(s) 232-245, ISSN 2329-0501
Publisher: lsevier
DOI: 10.1016/j.omtm.2019.08.014

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

Author(s): Juliane W. Schott, Diego León-Rico, Carolina B. Ferreira, Karen F. Buckland, Giorgia Santilli, Myriam A. Armant, Axel Schambach, Alessia Cavazza, Adrian J. Thrasher
Published in: Molecular Therapy - Methods & Clinical Development, Issue 14, 2019, Page(s) 134-147, ISSN 2329-0501
Publisher: Elsevier
DOI: 10.1016/j.omtm.2019.05.015

ESGCT 27th Annual Congress In collaboration with SETGyc Barcelona, Spain October 22–25, 2019 Abstracts

Author(s): M Zahn, C Lulay, S Afzal, R Fronza, W Wang, R Gabriel, B Gaspar, M Schmidt, I Gil-Farina
Published in: Human Gene Therapy, Issue 30/11, 2019, Page(s) A1-A221, ISSN 1043-0342
Publisher: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2019.29095.abstracts

Refining strategies to translate genome editing to the clinic

Author(s): Tatjana I Cornu, Claudio Mussolino, Toni Cathomen
Published in: Nature Medicine, Issue 23/4, 2017, Page(s) 415-423, ISSN 1078-8956
Publisher: Nature Publishing Group
DOI: 10.1038/nm.4313

Therapeutic genome editing with engineered nucleases

Author(s): S. A. Haas, V. Dettmer, T. Cathomen
Published in: Hämostaseologie, Issue 37/1, 2017, Page(s) 45-52, ISSN 0720-9355
Publisher: F. K. Schattauer Verlag
DOI: 10.5482/HAMO-16-09-0035

Multimodal Lentiviral Vectors for Pharmacologically Controlled Switching Between Constitutive Single Gene Expression and Tetracycline-Regulated Multiple Gene Collaboration

Author(s): Maike Stahlhut, Axel Schambach, Olga S. Kustikova
Published in: Human Gene Therapy Methods, Issue 28/4, 2017, Page(s) 191-204, ISSN 1946-6536
Publisher: Mary Ann Liebert Inc.
DOI: 10.1089/hgtb.2017.073

An optimized lentiviral vector system for conditional RNAi and efficient cloning of microRNA embedded short hairpin RNA libraries

Author(s): Felix F. Adams, Dirk Heckl, Thomas Hoffmann, Steven R. Talbot, Arnold Kloos, Felicitas Thol, Michael Heuser, Johannes Zuber, Axel Schambach, Adrian Schwarzer
Published in: Biomaterials, Issue 139, 2017, Page(s) 102-115, ISSN 0142-9612
Publisher: Pergamon Press Ltd.
DOI: 10.1016/j.biomaterials.2017.05.032

Viral and Synthetic RNA Vector Technologies and Applications

Author(s): Juliane W Schott, Michael Morgan, Melanie Galla, Axel Schambach
Published in: Molecular Therapy, Issue 24/9, 2016, Page(s) 1513-1527, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1038/mt.2016.143

Alpharetroviral self-inactivating vectors produced by a superinfection-resistant stable packaging cell line allow genetic modification of primary human T lymphocytes

Author(s): Verena Labenski, Julia D. Suerth, Elke Barczak, Dirk Heckl, Camille Levy, Ornellie Bernadin, Emmanuelle Charpentier, David A. Williams, Boris Fehse, Els Verhoeyen, Axel Schambach
Published in: Biomaterials, Issue 97, 2016, Page(s) 97-109, ISSN 0142-9612
Publisher: Pergamon Press Ltd.
DOI: 10.1016/j.biomaterials.2016.04.019

Using the CliniMACS PRODIGY® for CD34 enrichment and transduction of mobilised peripheral blood stem cells (mPBSC)

Author(s): E Armenteros-Monterroso1,2, K F Buckland2,1, A Diasakou1,2, I Pereira2,1, D Leon-Rico2,1, S Reinartz3, D Krenz3, U Bissels3, I Johnston3, E Papanikolaou3, C Booth1,2, A J Thrasher A J2,1
Published in: HUMAN GENE THERAPY, Issue Volume: 30 Issue: 11, 2019, Page(s) A89, ISSN 1043-0342
Publisher: Mary Ann Liebert Inc

Manufacture of an ATMP for the treatment of X-linked Severe Combined Immunodeficiency (X-SCID)

Author(s): Leon-Rico D, Schott JW, Armenteros-Monterroso E, Buckland KF, Diasakou A, Pereira I, Ferreira CB, Cavazza A, Shaw KL, Armant M, Kohn DB, Pai SY, Thasher AJ, Booth C
Published in: HUMAN GENE THERAPY, Issue Volume: 30 Issue: 11, 2019, Page(s) A86, ISSN 1043-0342
Publisher: Mary Ann Liebert, Inc

Intellectual Property Rights

Method for generating T-cell progenitors

Application/Publication number: WO 2018/146297
Date: 2018-08-16