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

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.

Field of science

  • /natural sciences/biological sciences/molecular biology/molecular evolution
  • /medical and health sciences/health sciences/public and environmental health/epidemics prevention/immunisation
  • /social sciences/sociology/demography/mortality
  • /medical and health sciences/basic medicine/pharmacology and pharmacy/pharmaceutical drug
  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

VRIJE UNIVERSITEIT BRUSSEL
Address
Pleinlaan 2
1050 Brussel
Belgium
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 101 370

Participants (7)

SORBONNE UNIVERSITE
France
EU contribution
€ 555 175
Address
21 Rue De L'ecole De Medecine
75006 Paris
Activity type
Higher or Secondary Education Establishments
ASSOCIATION GENETHON
France
EU contribution
€ 1 437 162,50
Address
Rue De L Internationale 1 Bis
91002 Evry
Activity type
Research Organisations
UNIVERSITATSKLINIKUM HEIDELBERG
Germany
EU contribution
€ 515 250
Address
Im Neuenheimer Feld 672
69120 Heidelberg
Activity type
Higher or Secondary Education Establishments
UNIVERSIDAD AUTONOMA DE BARCELONA
Spain
EU contribution
€ 491 730
Address
Calle Campus Universitario Sn Cerdanyola V
08290 Cerdanyola Del Valles
Activity type
Higher or Secondary Education Establishments
ENVIGO CRS LIMITED
United Kingdom
EU contribution
€ 1 156 500
Address
Woolley Road Alconbury
PE28 4HS Huntingdon
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
ASPHALION SL
Spain
EU contribution
€ 275 000
Address
Calle Tarragona, Numeros 151-157, Planta 10, Bloqu
08014 Barcelona
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH
Germany
EU contribution
€ 466 750
Address
Heinrich-hertz-allee 1
66386 St Ingbert
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)