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
- medical and health sciencesmedical biotechnologygenetic engineeringgene therapy
- medical and health sciencesclinical medicineoncologylung cancer
- natural sciencesbiological sciencescell biology
- medical and health sciencesmedical biotechnologycells technologiesstem cells
- medical and health scienceshealth sciencesinfectious diseasesRNA virusescoronaviruses
Programme(s)
Funding Scheme
SME-1 - SME instrument phase 1Coordinator
E91D9N2 Clonmel Tipperary
Ireland
The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.