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CureCN SIGNED

Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome

Total Cost €

EC-Contrib. €

Partnership

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Outcomes and
results

CureCN project word cloud

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

removal aav8 issue 1a1 small severe goals proposes constitutes valorization curative selective mutations authorization groundbreaking syndrome data recessive strategy diseases threatening diphosphate multicenter transgene additional broaden immunomodulatory bloodstream patient itself life technologies trial disease adeno carry treatment anti society 2020 strategies property intellectual cn virus safety burden transfer clinicians efficacy receiving treatments nabs cure prove economic validates proof najjar curecn rare rationale proportion antibodies gene translation therapy crigler label seropositive families expressing clinical medium aav irdirc additionally ultimately scope re disorder vector liver caused mediated marketing neutralizing uridine glucuronosyltransferase administration prevent patients ugt1a1 enterprises file scientists

Project "CureCN" data sheet

The following table provides information about the project.

Coordinator	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.
Coordinator Country	France [FR]
Project website	https://curecn.eu/
Total cost	6˙249˙103 €
EC max contribution	6˙249˙103 € (100%)
Programme	1. H2020-EU.3.1.3. (Treating and managing disease)
Code Call	H2020-SC1-2017-Two-Stage-RTD
Funding Scheme	RIA
Starting year	2018
Duration (year-month-day)	from 2018-01-01 to 2022-12-31

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	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)	coordinator	3˙738˙478.00
2	ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM Organization address address: MEIBERGDREEF 15 city: AMSTERDAM postcode: 1105AZ website: www.amc.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 (AMSTERDAM)	participant	526˙718.00
3	EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH Organization address address: HEINRICH-HERTZ-ALLEE 1 city: ST INGBERT postcode: 66386 website: www.eurice.eu contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DE (ST INGBERT)	participant	516˙125.00
4	UNIVERSITY OF LEICESTER UNIVERSITY OF LEICESTER Organization address address: UNIVERSITY ROAD city: LEICESTER postcode: LE1 7RH website: www.le.ac.uk contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (LEICESTER)	participant	396˙182.00
5	GENOSAFE SAS GENOSAFE SAS Organization address address: RUE DE L'INTERNATIONALE 1 city: EVRY postcode: 91000 website: www.genosafe.com 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	207˙017.00
6	ASSISTANCE PUBLIQUE HOPITAUX DE PARIS ASSISTANCE PUBLIQUE HOPITAUX DE PARIS Organization address address: 3 AVENUE VICTORIA city: PARIS postcode: 75000 website: http://www.aphp.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	172˙083.00
7	MC TOXICOLOGY CONSULTING GMBH MC TOXICOLOGY CONSULTING GMBH Organization address address: SIEBENSTERNGASSE 31/8 city: WIEN postcode: 1070 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.	AT (WIEN)	participant	156˙872.00
8	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	149˙000.00
9	AZIENDA OSPEDALIERA PAPA GIOVANNI XXIII AZIENDA OSPEDALIERA PAPA GIOVANNI XXIII Organization address address: PIAZZA OMS 1 city: BERGAMO postcode: 24127 website: www.ospedaliriuniti.bergamo.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 (BERGAMO)	participant	147˙500.00
10	UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II Organization address address: CORSO UMBERTO I, 40 city: NAPOLI postcode: 80138 website: www.unina.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 (NAPOLI)	participant	147˙125.00
11	ASSOCIATION FRANCAISE DE CRIGLER NAJJAR ASSOCIATION FRANCAISE DE CRIGLER NAJJAR Organization address address: 8 RUE HENRI GOLAUDIN city: CLAMART postcode: 92140 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.	FR (CLAMART)	participant	92˙000.00

Map

Project objective

Crigler-Najjar syndrome (CN) is a rare recessive disorder caused by mutations in the uridine diphosphate glucuronosyltransferase 1A1(UGT1A1) gene. CN is a life-threatening disease with no cure which constitutes a severe burden for the patients, their families, and the society. CureCN has the objective of developing a curative gene therapy for CN syndrome based on liver gene transfer with and adeno-associated virus (AAV) vector expressing the UGT1A1 transgene. Additional goals of CureCN are to develop strategies to allow for vector re-administration and to address the issue of pre-existing anti-AAV neutralizing antibodies (NAbs), which prevent large proportion of seropositive patients from receiving AAV mediated gene therapy. Proof-of-concept studies of AAV8-UGT1A1 gene transfer provide a strong rationale for the safety and efficacy of gene therapy for CN. CureCN proposes to carry out an open-label, multicenter clinical trial of AAV8-UGT1A1 gene transfer to prove the safety and efficacy of the therapy in severe CN patients, and file for marketing authorization in Europe at the end of the study. CureCN will also produce enabling data for the clinical translation of a groundbreaking immunomodulatory strategy to allow for vector administration. Additionally, a technology for the selective removal of anti-AAV NAbs from the bloodstream of seropositive patients will be developed. The goal of these studies is to ultimately allow all CN patients to access AAV8-UGT1A1 gene therapy. CureCN is a patient-driven initiative that gathers top clinicians and scientists; it also includes small medium enterprises in its partners, to foster economic growth and valorization of intellectual property. CureCN sets itself in the ambitious goal set by the IRDiRC by 2020 by developing a curative treatment for CN syndrome. Importantly, it validates technologies that will broaden the scope of gene therapy, thus will have an impact on the development of treatments for several other rare diseases.

Deliverables

List of deliverables.
Public project website	Websites, patent fillings, videos etc.	2020-02-18 10:25:17

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

Publications

List of publications.
year	authors and title	journal	last update
2019	Fanny Collaud, Giulia Bortolussi, Laurence Guianvarcâ€™h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome published pages: 157-174, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.12.011	Molecular Therapy - Methods & Clinical Development 12	2020-02-18

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