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Development of an innovative gene therapy platform to cure rare hereditary muscle disorders

Total Cost €


EC-Contrib. €






 MYOCURE project word cloud

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

stakeholders    disorder    preclinical    minimize    economic    modified    provoke    trial    aav    de    mtm    computationally    rare    scalable    skeletal    models    storage    suffering    efficient    subsequent    irdirc    innovative    model    basis    cardiac    validated    cure    boosting    lower    dysfunction    platform    hereditary    myopathy    family    gsd    genetic    bottlenecks    200    clinical    glycogen    disease    therapeutic    maximize    diaphragm    adeno    constitute    fold    proteins    molecular    2020    immune    medical    mortality    doses    diseases    hamper    expression    animal    reactions    caused    myotubular    suboptimal    therapies    safer    cells    phc14    cures    myocure    manufacturing    therapy    consolidate    400    consistent    patients    drug    viral    designation    muscle    transfer    single    roadmap    attractive    evolution    inherited    vector    novo    disorders    compromise    designed    atmp    vectors    treatment    diverse    gene    orphan    safety    defects    toxicology    morbidity    efficacy    overcoming    outcome    promoters    option    directed    applies    undesirable   

Project "MYOCURE" data sheet

The following table provides information about the project.


Organization address
address: PLEINLAAN 2
postcode: 1050

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 Belgium [BE]
 Project website
 Total cost 5˙998˙937 €
 EC max contribution 5˙998˙937 € (100%)
 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    VRIJE UNIVERSITEIT BRUSSEL BE (BRUSSEL) coordinator 1˙101˙370.00
2    ASSOCIATION GENETHON FR (EVRY) participant 1˙437˙162.00
3    ENVIGO CRS LIMITED UK (HUNTINGDON) participant 1˙156˙500.00
4    SORBONNE UNIVERSITE FR (PARIS) participant 555˙175.00
8    ASPHALION SL ES (BARCELONA) participant 275˙000.00


 Project objective

The objective of MYOCURE is to develop an innovative gene therapy platform to cure rare hereditary muscle disorders, specifically focusing on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II. These are attractive diseases for gene therapy since they compromise a diverse family of rare genetic diseases typically caused by single gene defects that often provoke significant morbidity and mortality due to skeletal muscle, cardiac and/or diaphragm dysfunction. There are no effective cures and current treatment is suboptimal. MYOCURE focuses specifically on overcoming the key bottlenecks that hamper muscle-directed gene therapy by (i) boosting gene transfer using muscle-specific adeno-associated viral vectors (AAV) generated de novo by directed molecular evolution (ii) increasing expression by using robust computationally designed muscle-specific promoters that are 400-fold more efficient than the state of the art, allowing the use of lower and thus safer therapeutic vector doses (iii) minimize undesirable immune reactions against the vector, gene-modified muscle cells and therapeutic proteins. The efficacy and safety of this advanced therapy medical product (ATMP) will be validated in preclinical MTM and GSD II animal models. A scalable manufacturing process will be developed for subsequent toxicology studies and an orphan drug designation will be applied for. MYOCURE will consolidate a roadmap towards clinical development and economic valorisation and maximize dissemination to the relevant stakeholders. The outcome of MYOCURE will constitute the basis of a Phase I gene therapy clinical trial in MTM and GSD patients consistent with the IRDiRC objectives to provide 200 therapies for rare disease by 2020. MYOCURE applies to 'PHC14 New Therapies for Rare Diseases' and advances the development of a new therapeutic option for patients suffering from these rare inherited muscle diseases, as well as related preclinical research and animal model development.


List of deliverables.
Project website Websites, patent fillings, videos etc. 2020-01-30 08:49:24

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


year authors and title journal last update
List of publications.
2019 Warut Tulalamba, Jonas Weinmann, Quang Hong Pham, Jihad El Andari, Thierry VandenDriessche, Marinee K. Chuah, Dirk Grimm
Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors
published pages: , ISSN: 0969-7128, DOI: 10.1038/s41434-019-0106-3
Gene Therapy 2020-03-05
2019 S. Sarcar, W. Tulalamba, M. Y. Rincon, J. Tipanee, H. Q. Pham, H. Evens, D. Boon, E. Samara-Kuko, M. Keyaerts, M. Loperfido, E. Berardi, S. Jarmin, P. In’t Veld, G. Dickson, T. Lahoutte, M. Sampaolesi, P. De Bleser, T. VandenDriessche, M. K. Chuah
Next-generation muscle-directed gene therapy by in silico vector design
published pages: , ISSN: 2041-1723, DOI: 10.1038/s41467-018-08283-7
Nature Communications 10/1 2020-03-05
2017 Maria Luisa Jaén, Laia Vilà, Ivet Elias, Veronica Jimenez, Jordi Rodó, Luca Maggioni, Rafael Ruiz-de Gopegui, Miguel Garcia, Sergio Muñoz, David Callejas, Eduard Ayuso, Tura Ferré, Iris Grifoll, Anna Andaluz, Jesus Ruberte, Virginia Haurigot, Fatima Bosch
Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs
published pages: 1-7, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2017.03.008
Molecular Therapy - Methods & Clinical Development 6 2020-01-30
2017 Francesco Puzzo, Pasqualina Colella, Maria G. Biferi, Deeksha Bali, Nicole K. Paulk, Patrice Vidal, Fanny Collaud, Marcelo Simon-Sola, Severine Charles, Romain Hardet, Christian Leborgne, Amine Meliani, Mathilde Cohen-Tannoudji, Stephanie Astord, Bernard Gjata, Pauline Sellier, Laetitia van Wittenberghe, Alban Vignaud, Florence Boisgerault, Martine Barkats, Pascal Laforet, Mark A. Kay, Dwight D. Koeberl, Giuseppe Ronzitti, Federico Mingozzi
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
published pages: eaam6375, ISSN: 1946-6234, DOI: 10.1126/scitranslmed.aam6375
Science Translational Medicine 9/418 2020-01-30
2017 Dirk Grimm, Hildegard Büning
Small But Increasingly Mighty: Latest Advances in AAV Vector Research, Design, and Evolution
published pages: 1075-1086, ISSN: 1043-0342, DOI: 10.1089/hum.2017.172
Human Gene Therapy 28/11 2020-01-30
2018 Veronica Jimenez, Claudia Jambrina, Estefania Casana, Victor Sacristan, Sergio Muñoz, Sara Darriba, Jordi Rodó, Cristina Mallol, Miquel Garcia, Xavier León, Sara Marcó, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura Ferré, Sandra Motas, Sylvie Franckhauser, Francisca Mulero, Marc Navarro, Virginia Haurigot, Jesus Ruberte, Fatima Bosch
FGF21 gene therapy as treatment for obesity and insulin resistance
published pages: e8791, ISSN: 1757-4676, DOI: 10.15252/emmm.201708791
EMBO Molecular Medicine 10/8 2020-01-30
2019 Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, Francesco Puzzo, Laetitia van Wittenberghe, Nicolas Guerchet, Nathalie Daniele, Bernard Gjata, Solenne Marmier, Severine Charles, Marcelo Simon Sola, Isabella Ragone, Christian Leborgne, Fanny Collaud, Federico Mingozzi
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
published pages: 85-101, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.11.002
Molecular Therapy - Methods & Clinical Development 12 2020-01-30
2018 Amine Meliani, Florence Boisgerault, Romain Hardet, Solenne Marmier, Fanny Collaud, Giuseppe Ronzitti, Christian Leborgne, Helena Costa Verdera, Marcelo Simon Sola, Severine Charles, Alban Vignaud, Laetitia van Wittenberghe, Giorgia Manni, Olivier Christophe, Francesca Fallarino, Christopher Roy, Alicia Michaud, Petr Ilyinskii, Takashi Kei Kishimoto, Federico Mingozzi
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration
published pages: , ISSN: 2041-1723, DOI: 10.1038/s41467-018-06621-3
Nature Communications 9/1 2020-01-30
2018 Klaudia Kuranda, Priscilla Jean-Alphonse, Christian Leborgne, Romain Hardet, Fanny Collaud, Solenne Marmier, Helena Costa Verdera, Giuseppe Ronzitti, Philippe Veron, Federico Mingozzi
Exposure to wild-type AAV drives distinct capsid immunity profiles in humans
published pages: 5267-5279, ISSN: 0021-9738, DOI: 10.1172/jci122372
Journal of Clinical Investigation 128/12 2020-01-30

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