Project description
Development of innovative therapies for rare neuromuscular disorders
Currently, less than 6 % of rare diseases have approved therapies, and the World Health Organization highlights a critical need for new treatments. The EU-funded DREAMS project will address this by producing therapies for five rare neuromuscular disorders with similar pathophysiological traits. The project's innovative approach involves using skeletal muscle cells from induced pluripotent stem cells to discover common biomarkers across these disorders. Additionally, it employs AI to identify shared therapeutic targets and potential drug candidates from phenotypic drug screenings and -omics data. The resulting platform will combine AI and induced pluripotent stem cells to develop small molecule treatments applicable to multiple neuromuscular disorders, setting a new standard in rare disease therapy research.
Objective
According to World Health Organization, to date, fewer than 6% of rare diseases (RDs) have an approved treatment option. In the light of this observation, it appears crucial to develop disruptive approaches that allow to find treatments for multiple RDs. To achieve this goal, DREAMS will start from 5 rare neuromuscular disorders (NMD) that share common pathophysiological characteristics related to dysfunctions of autophagy and desmin disorganization. Using skeletal muscle cells derived from induced pluripotent stem cells (skMC-iPSC), DREAMS will i) identify shared biomarkers of the 5 diseases and ii) perform a high throughput drug screen on all 5 diseases. Through a first Artificial Intelligence (AI) based solution, the output of the drug screening will be used to find drug targets shared between the diseases. A second AI based solution will be used to find drug candidates (repurposable drugs and new molecular entities) for these shared drug targets. A third AI-based solution will be used to find additional diseases that could be treated through the shared drug targets, in order to extend the indications for the most promising drug candidates. Following their discoveries, the promising drug candidates will be tested for efficacy and safety in both iPSC and mouse models of the 5 NMDs and additional diseases. In parallel, DREAMS will design an adaptive clinical trial to prepare the final stages of drug development of these novel drug candidates with multiple indications. Together, these technological and conceptual innovations will allow the DREAMS consortium to i) develop therapies to treat multiple NMDs and related diseases, ii) to create a novel clinical design that generates a regulatory pathway for drugs that treat heterogeneous groups of patients suffering from RDs iii) to further the scientific understanding of commonalities in RDs, and iv) to create a reusable drug development platform to discover safe and effective treatments for RDs in general.
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.2.1 - Health
MAIN PROGRAMME
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HORIZON.2.1.3 - Non-Communicable and Rare Diseases
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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-RIA - HORIZON Research and Innovation Actions
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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-HLTH-2022-DISEASE-06-two-stage
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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.
91100 Corbeil-Essonnes
France
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.