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

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.

Coordinator

VRIJE UNIVERSITEIT BRUSSEL

Address

Pleinlaan 2
1050 Brussel

Belgium

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 101 370

Participants (8)

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UNIVERSITE PIERRE ET MARIE CURIE - PARIS 6

France

SORBONNE UNIVERSITE

France

EU Contribution

€ 555 175

ASSOCIATION GENETHON

France

EU Contribution

€ 1 437 162,50

UNIVERSITATSKLINIKUM HEIDELBERG

Germany

EU Contribution

€ 515 250

UNIVERSITAT AUTONOMA DE BARCELONA

Spain

EU Contribution

€ 491 730

ENVIGO CRS LIMITED

United Kingdom

EU Contribution

€ 1 156 500

ASPHALION SL

Spain

EU Contribution

€ 275 000

EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH

Germany

EU Contribution

€ 466 750

Project information

Grant agreement ID: 667751

Status

Ongoing project

  • Start date

    1 January 2016

  • End date

    31 December 2019

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 998 937,50

  • EU contribution

    € 5 998 937,50

Coordinated by:

VRIJE UNIVERSITEIT BRUSSEL

Belgium