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MYOCURE SIGNED

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

Total Cost €

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EC-Contrib. €

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Partnership

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Outcomes and
results

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.

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

Project "MYOCURE" data sheet

The following table provides information about the project.

Coordinator	VRIJE UNIVERSITEIT BRUSSEL Organization address address: PLEINLAAN 2 city: BRUSSEL postcode: 1050 website: www.vub.ac.be 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	http://www.myocure.eu
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

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	VRIJE UNIVERSITEIT BRUSSEL VRIJE UNIVERSITEIT BRUSSEL Organization address address: PLEINLAAN 2 city: BRUSSEL postcode: 1050 website: www.vub.ac.be contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	BE (BRUSSEL)	coordinator	1˙101˙370.00
2	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	1˙437˙162.00
3	ENVIGO CRS LIMITED ENVIGO CRS LIMITED Organization address address: WOOLLEY ROAD ALCONBURY city: HUNTINGDON postcode: PE28 4HS website: www.huntingdon.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (HUNTINGDON)	participant	1˙156˙500.00
4	SORBONNE UNIVERSITE SORBONNE UNIVERSITE Organization address address: 21 RUE DE L'ECOLE DE MEDECINE city: PARIS postcode: 75006 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 (PARIS)	participant	555˙175.00
5	UNIVERSITATSKLINIKUM HEIDELBERG UNIVERSITATSKLINIKUM HEIDELBERG Organization address address: IM NEUENHEIMER FELD 672 city: HEIDELBERG postcode: 69120 website: www.klinikum.uni-heidelberg.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 (HEIDELBERG)	participant	515˙250.00
6	UNIVERSIDAD AUTONOMA DE BARCELONA UNIVERSIDAD AUTONOMA DE BARCELONA Organization address address: CALLE CAMPUS UNIVERSITARIO SN CERDANYOLA V city: CERDANYOLA DEL VALLES postcode: 8290 website: http://www.uab.es contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (CERDANYOLA DEL VALLES)	participant	491˙730.00
7	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	466˙750.00
8	ASPHALION SL ASPHALION SL Organization address address: CALLE TARRAGONA, NUMEROS 151-157, PLANTA 10, BLOQU city: BARCELONA postcode: 8014 website: www.asphalion.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (BARCELONA)	participant	275˙000.00
9	UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6 UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6 Organization address address: Place Jussieu 4 city: PARIS postcode: 75252 website: www.upmc.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	0.00

Map

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.

Deliverables

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.

Publications

List of publications.
year	authors and title	journal	last update
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. Inâ€™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 JaÃ©n, Laia VilÃ , Ivet Elias, Veronica Jimenez, Jordi RodÃ³, Luca Maggioni, Rafael Ruiz-de Gopegui, Miguel Garcia, Sergio MuÃ±oz, David Callejas, Eduard Ayuso, Tura FerrÃ©, 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 Î±-glucosidase published pages: eaam6375, ISSN: 1946-6234, DOI: 10.1126/scitranslmed.aam6375	Science Translational Medicine 9/418	2020-01-30
2017	Dirk Grimm, Hildegard BÃ¼ning 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 MuÃ±oz, Sara Darriba, Jordi RodÃ³, Cristina Mallol, Miquel Garcia, Xavier LeÃ³n, Sara MarcÃ³, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura FerrÃ©, 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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