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SCIDNET SIGNED

DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID)

Total Cost €

EC-Contrib. €

Partnership

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Outcomes and
results

SCIDNET project word cloud

Explore the words cloud of the SCIDNET project. It provides you a very rough idea of what is the project "SCIDNET" about.

infants orphan genetic innovative option involvement normal though ready unless of medicines children clinical irdirc agencies whereby combined act lives die medicinal drug trial ec 80 2000 scid authorised technologies therapy 141 agency limited engagement live translation options trials devastating life proof licensed ambitions treatment members allogeneic regulation last autologous marketing criteria bone 15 assisted disease gene severe scidnet paradigm shown authorisation position correcting rare functional curative first disorder born medicine ada safety cell classified haematopoietic stem x1 immune unmet disorders regulatory immunodeficiency efficacy transplantation designation marrow forms keeping

Project "SCIDNET" data sheet

The following table provides information about the project.

Coordinator	UNIVERSITY COLLEGE LONDON Organization address address: GOWER STREET 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	https://scidnet.eu/
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

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	UNIVERSITY COLLEGE LONDON UNIVERSITY COLLEGE LONDON Organization address address: GOWER STREET 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.	UK (LONDON)	coordinator	1˙549˙174.00
2	INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE Organization address address: RUE DE TOLBIAC 101 city: PARIS postcode: 75654 website: www.inserm.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (PARIS)	participant	850˙002.00
3	ASSOCIATION GENETHON ASSOCIATION GENETHON Organization address address: RUE DE L INTERNATIONALE 1 BIS city: EVRY postcode: 91002 website: www.genethon.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (EVRY)	participant	800˙000.00
4	ACADEMISCH ZIEKENHUIS LEIDEN ACADEMISCH ZIEKENHUIS LEIDEN Organization address address: ALBINUSDREEF 2 city: LEIDEN postcode: 2333 ZA website: www.lumc.nl contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	NL (LEIDEN)	participant	799˙877.00
5	GENEWERK GMBH GENEWERK GMBH Organization address address: IM NEUENHEIMER FELD 582 city: HEIDELBERG postcode: 69120 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.	DE (HEIDELBERG)	participant	699˙750.00
6	MEDIZINISCHE HOCHSCHULE HANNOVER MEDIZINISCHE HOCHSCHULE HANNOVER Organization address address: Carl-Neuberg-Strasse 1 city: HANNOVER postcode: 30625 website: www.mh-hannover.de contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DE (HANNOVER)	participant	509˙562.00
7	UNIVERSITAETSKLINIKUM FREIBURG UNIVERSITAETSKLINIKUM FREIBURG Organization address address: HUGSTETTER STRASSE 49 city: FREIBURG postcode: 79106 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.	DE (FREIBURG)	participant	499˙300.00
8	OSPEDALE SAN RAFFAELE SRL OSPEDALE SAN RAFFAELE SRL Organization address address: VIA OLGETTINA 60 city: MILANO postcode: 20132 website: www.hsr.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (MILANO)	participant	474˙250.00
9	GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST Organization address address: GREAT ORMOND STREET city: LONDON postcode: WC1N 3JH 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.	UK (LONDON)	participant	423˙198.00
10	MILTENYI BIOTEC GMBH MILTENYI BIOTEC GMBH Organization address address: FRIEDRICH EBERT STRASSE 68 city: BERGISH GLADBACH postcode: 51429 website: www.miltenyibiotec.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DE (BERGISH GLADBACH)	participant	296˙200.00
11	INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES Organization address address: MAIN ROAD FIRSIDE city: TORPOINT postcode: PL11 3LE 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.	UK (TORPOINT)	participant	25˙000.00
12	CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS Organization address address: RUE DU BUGNON 21 city: LAUSANNE postcode: 1011 website: www.chuv.ch contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	CH (LAUSANNE)	participant	0.00

Map

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.

Deliverables

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.

Publications

List of publications.
year	authors and title	journal	last update
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 PRODIGYÂ® 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, GrÃ©gory CÃ©drone, 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 OlivrÃ©, Sandrine Susini, Kevin Appourchaux, Christian Reimann, Hanem Sadek, Olivier PellÃ©, Nicolas Cagnard, Elisa Magrin, Chantal Lagresle-Peyrou, Tom Taghon, Antonio Rausell, Marina Cavazzana, Isabelle AndrÃ©-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 BÃ¼ning, 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 LeÃ³n-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 LÃ©vy, Kuiying Ma, Caroline Costa, Didier NÃ¨gre, Christian Reimann, David Fenard, Agata Cieslak, Vahid Asnafi, Hanem Sadek, Rana Mhaidly, Marina Cavazzana, Chantal Lagresle-Peyrou, FranÃ§ois-LoÃ¯c Cosset, Isabelle AndrÃ©, 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 22â€“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, NicolÃ² 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 â€“ 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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