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DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID)

Total Cost €


EC-Contrib. €






Project "SCIDNET" data sheet

The following table provides information about the project.


Organization address
city: LONDON
postcode: WC1E 6BT
website: n.a.

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country United Kingdom [UK]
 Project website
 Total cost 7˙474˙316 €
 EC max contribution 6˙926˙313 € (93%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-PHC-2015-two-stage
 Funding Scheme RIA
 Starting year 2016
 Duration (year-month-day) from 2016-01-01   to  2019-12-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITY COLLEGE LONDON UK (LONDON) coordinator 1˙549˙174.00
3    ASSOCIATION GENETHON FR (EVRY) participant 800˙000.00
4    ACADEMISCH ZIEKENHUIS LEIDEN NL (LEIDEN) participant 799˙877.00
5    GENEWERK GMBH DE (HEIDELBERG) participant 699˙750.00
8    OSPEDALE SAN RAFFAELE SRL IT (MILANO) participant 474˙250.00
10    MILTENYI BIOTEC GMBH DE (BERGISH GLADBACH) participant 296˙200.00


 Project objective

Severe combined immunodeficiency (SCID) is a devastating rare disorder of immune system development. Affected infants are born without functional immune systems and die within the first year of life unless effective treatment is given. Treatment options are limited to allogeneic haematopoietic stem cell transplantation and autologous stem cell gene therapy. Over the last 15 years, gene therapy for two forms of SCID (SCID-X1 and ADA SCID) has shown significant safety and efficacy in correcting the immunodeficiency and allowing children to live normal lives. Proof of concept of gene therapy for 3 other SCID forms has also been shown by members of the proposed SCIDNET consortium and is ready for translation into clinical trials. We are therefore in a position whereby, over the next 4 years, we can offer gene therapy as a curative option for over 80% of all forms of SCID in Europe. Importantly for 1 of these conditions (ADA SCID) we will undertake clinical trials that will lead to marketing authorisation of the gene therapy product as a licensed medicine. In addition, we will investigate the future technologies that will improve the safety and efficacy of gene therapy for SCID.

Our proposal addresses an unmet clinical need in SCID, which is classified as a rare disease according to EU criteria (EC regulation No. 141/2000). The proposal also addresses the need to develop an innovative treatment such as gene therapy from early clinical trials though to a licensed medicinal product through involvement with regulatory agencies and is in keeping with the ambitions of the IRDiRC. The lead ADA SCID programme has Orphan Drug Designation and clinical trial design is assisted by engagement with the European medicines Agency. The ADA SCID trial will act as a paradigm for the development of the technologies and processes that will allow gene therapy for not only SCID, but also other bone marrow disorders, to become authorised genetic medicines in the future.


List of deliverables.
A set of clinically applicable vectors that can correct RAG1 and RAG2 deficiency in mouse models and human cells Documents, reports 2020-04-24 23:02:51
Gene editing protocol for one disease entity Documents, reports 2020-04-24 23:03:13
Minutes of third SAB Documents, reports 2020-04-24 23:03:25
Correct OS in mouse model and patient cells Documents, reports 2020-04-24 23:03:03
Efficacy of Artemis clinical batch and ODD status Documents, reports 2020-04-24 23:03:37
Minutes of second SAB Documents, reports 2020-04-14 21:21:20
Project kick-off meeting minutes Documents, reports 2020-04-14 21:21:19
Website for SCIDNET Documents, reports 2020-04-14 21:21:19
Setting up of SCIDNET project website Demonstrators, pilots, prototypes 2020-04-14 21:21:19
Minutes of 1st General Assembly meeting Documents, reports 2020-04-14 21:21:19
Minutes of first SAB Documents, reports 2020-04-14 21:21:19

Take a look to the deliverables list in detail:  detailed list of SCIDNET deliverables.


year authors and title journal last update
List of publications.
2019 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
Manufacture of an ATMP for the treatment of X-linked Severe Combined Immunodeficiency (X-SCID)
published pages: A86, ISSN: 1043-0342, DOI:
HUMAN GENE THERAPY Volume: 30 Issue: 11 2020-04-14
2019 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
Using the CliniMACS PRODIGY® for CD34 enrichment and transduction of mobilised peripheral blood stem cells (mPBSC)
published pages: A89, ISSN: 1043-0342, DOI:
HUMAN GENE THERAPY Volume: 30 Issue: 11 2020-04-14
2019 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
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
published pages: 137-150, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2018.10.008
Molecular Therapy 27/1 2020-04-14
2019 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
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
published pages: 232-245, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2019.08.014
Molecular Therapy - Methods & Clinical Development 15 2020-04-14
2018 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
Generation of adult human T-cell progenitors for immunotherapeutic applications
published pages: 1491-1494.e4, ISSN: 0091-6749, DOI: 10.1016/j.jaci.2017.10.034
Journal of Allergy and Clinical Immunology 141/4 2020-04-14
2018 Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency
published pages: 257-269, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.03.002
Molecular Therapy - Methods & Clinical Development 9 2020-04-14
2019 Denise Klatt, Erica Cheng, Friederike Philipp, Anton Selich, Julia Dahlke, Reinhold E. Schmidt, Juliane W. Schott, Hildegard Büning, Dirk Hoffmann, Adrian J. Thrasher, Axel Schambach
Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes
published pages: 590-598, ISSN: 2213-6711, DOI: 10.1016/j.stemcr.2019.08.008
Stem Cell Reports 13/4 2020-04-14
2019 Juliane W. Schott, Diego León-Rico, Carolina B. Ferreira, Karen F. Buckland, Giorgia Santilli, Myriam A. Armant, Axel Schambach, Alessia Cavazza, Adrian J. Thrasher
Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application
published pages: 134-147, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2019.05.015
Molecular Therapy - Methods & Clinical Development 14 2020-04-14
2019 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
Baboon envelope LVs efficiently transduced human adult, fetal, and progenitor T cells and corrected SCID-X1 T-cell deficiency
published pages: 461-475, ISSN: 2473-9529, DOI: 10.1182/bloodadvances.2018027508
Blood Advances 3/3 2020-04-14
2019 M Zahn, C Lulay, S Afzal, R Fronza, W Wang, R Gabriel, B Gaspar, M Schmidt, I Gil-Farina
ESGCT 27th Annual Congress In collaboration with SETGyc Barcelona, Spain October 22–25, 2019 Abstracts
published pages: A1-A221, ISSN: 1043-0342, DOI: 10.1089/hum.2019.29095.abstracts
Human Gene Therapy 30/11 2020-04-14
2018 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
Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation
published pages: 928-941.e8, ISSN: 0091-6749, DOI: 10.1016/j.jaci.2017.11.015
Journal of Allergy and Clinical Immunology 142/3 2020-04-14
2016 Kathryn V. Whitmore, Hubert B. Gaspar
Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency
published pages: , ISSN: 1664-3224, DOI: 10.3389/fimmu.2016.00314
Frontiers in Immunology 7 2020-04-14

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