Project description
Exosome-mediated gene therapy for cystic fibrosis
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CFTR gene, and it is characterised by persistent lung infections, limiting the breathing ability of patients. Gene therapy to replace the defective gene in the lungs has been proposed as the cure for CF. To deliver the gene into the lungs, the EU-funded ReSpire project proposes to use exosomes, cell-secreted vesicles used for intercellular communication. Scientists have engineered the exosome surface to achieve enhanced mucus penetration and cell transfection, while delivery to the lungs will be performed using a nebulisation aerosol method. The ReSpire approach has great potential to treat CF and other lung diseases with high unmet medical need, improving both survival rates and the quality of life of CF patients.
Objective
Via the SME Instrument OmniSpirant Ltd will mature its stem cell exosome-packaged gene therapy with lung mucus
penetration properties which will be more effective (improved lung function, reversal of lung damage and superior CFTR
gene expression and function) and also be applicable to 100% of the CF global population. This approach, using a combined
patent pending exosome surface engineering for enhanced mucus penetration and cell transfection with a tailored vibrating
mesh nebulisation aerosol delivery method will provide a unique, potentially disruptive, therapeutic modality for CF and many
other lung diseases with high unmet medical need.
Our competitive advantage and Unique Selling Points lower current effectiveness barriers and make the OmniSpirant
exosome technology the only therapeutic delivery method that could provide a truly effective treatment for CF, thus
improving both survival rates the quality of life of CF suffers. Importantly, the quality of life for CF patients will increase to
point where they are able to work. The OmniSpirant system will, over time, be regarded as an essential weapon in the
armoury of health systems in the battle against other serious respiratory conditions affecting major patient populations such
as lung cancer and COPD, globally.
The overall objective of our feasibility study is to demonstrate the potential value of the combined gene therapy system
maturation based on a comprehensive analysis of the business concept in terms of technical, commercial and financial
viability. The outcome of the study will be a full Business Plan that will feed into a Phase 2 project.
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.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences clinical medicine oncology lung cancer
- medical and health sciences clinical medicine physiotherapy
- medical and health sciences medical biotechnology cells technologies stem cells
- medical and health sciences health sciences infectious diseases RNA viruses coronaviruses
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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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H2020-EU.2.3. - INDUSTRIAL LEADERSHIP - Innovation In SMEs
MAIN PROGRAMME
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H2020-EU.3. - PRIORITY 'Societal challenges
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H2020-EU.2.1. - INDUSTRIAL LEADERSHIP - Leadership in enabling and industrial technologies
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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.
SME-1 - SME instrument phase 1
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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) H2020-EIC-SMEInst-2018-2020
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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.
E91D9N2 Clonmel Tipperary
Ireland
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.