Objective
Muscular dystrophies (MDs) are a group of about 50 different monogenic disorders characterized by progressive, irreversible muscle wasting, leading to loss of autonomy and premature death. Patients rely on palliative care; the disease burden is immense. Current gene therapy approaches do not address the underlying genetic causes. The challenges to develop gene therapies for MDs include the genetic variability and the lack of a safe drug delivery methods to muscle.
MyoPax is a clinic-ready company focused on restoring muscle function in patients with muscle diseases via targeted regenerative therapies. For genetic MDs we have developed the Satgeno platform: We aim to harness the body’s natural regenerative capabilities to sustainably deliver a gene repair therapy for the first time to muscle. Satgeno leverages our patented stem cell technology to manufacture highly regenerative muscle stem (“satellite”) cells called Primary Human Satellite cells-derived muscle stem cells (PHSats). PHSats rebuild muscle tissue and, very importantly, generate new muscle stem cells in their respective niche. This capability allows us to 1) correct MD-causing mutations ex vivo in PHSats using virus-free, mRNA-mediated, CRISPR-derived gene editing, 2) deliver gene-corrected healthy stem cells to functionally important muscles in MD patients, and 3) sustainably rebuild healthy muscle function.
With EIC support, we will conduct the first-in-human DELI550 trial, focusing on a frequent founder mutation. Pending safety and efficacy validation, we expand our approach into a comprehensive platform to treat various MD mutations affecting 400K+ people in the EU and US. This trial will mark a breakthrough. The platform has the potential to transform the patient´s treatment as acknowledged by Nature medicine “11 Clinical Trials that Will Shape Medicine in 2023”. We strive for a future where individuals with MDs experience significantly improved quality of life and functional independence.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- engineering and technology mechanical engineering vehicle engineering aerospace engineering satellite technology
- medical and health sciences medical biotechnology cells technologies stem cells
- natural sciences biological sciences genetics mutation
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
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HORIZON.3.1 - The European Innovation Council (EIC)
MAIN PROGRAMME
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Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
HORIZON-EIC-ACC - HORIZON EIC Accelerator
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Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) HORIZON-EIC-2024-ACCELERATOR-02
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Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.
10115 BERLIN
Germany
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.