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CureCN SIGNED

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

Total Cost €

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EC-Contrib. €

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Partnership

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 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.

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

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 FR (EVRY) coordinator 3˙738˙478.00
2    ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM NL (AMSTERDAM) participant 526˙718.00
3    EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH DE (ST INGBERT) participant 516˙125.00
4    UNIVERSITY OF LEICESTER UK (LEICESTER) participant 396˙182.00
5    GENOSAFE SAS FR (EVRY) participant 207˙017.00
6    ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR (PARIS) participant 172˙083.00
7    MC TOXICOLOGY CONSULTING GMBH AT (WIEN) participant 156˙872.00
8    MEDIZINISCHE HOCHSCHULE HANNOVER DE (HANNOVER) participant 149˙000.00
9    AZIENDA OSPEDALIERA PAPA GIOVANNI XXIII IT (BERGAMO) participant 147˙500.00
10    UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II IT (NAPOLI) participant 147˙125.00
11    ASSOCIATION FRANCAISE DE CRIGLER NAJJAR 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

year authors and title journal last update
List of publications.
2019 Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’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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The information about "CURECN" are provided by the European Opendata Portal: CORDIS opendata.

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