Periodic Reporting for period 1 - ReSpire (Respirable Advanced Therapeutics for Cystic Fibrosis & other Lung Diseases)
Reporting period: 2019-02-01 to 2019-07-31
Current CF therapies involve chronic use of suppressive antibiotic therapy allied with use of mucolytics to reduce the viscous secretions and anti-inflammatory agents, bronchodilators and inhaled steroids to ease breathing plus physiotherapy to actively help removal of mucus. Some patients may also benefit by adding in new CFTR modulator drugs. CF gene therapy to replace the mutated CFTR gene with a healthy copy would be an ideal treatment, the concept was proven over 20 years ago in simple cell models but has failed to show clinical study benefits to date.
The problem is that the thick mucus in the lungs of CF sufferers is a barrier that traps the carriers used to deliver inhaled CFTR gene therapies such as nanoparticles and viral vectors. These trapped gene therapy carriers are mostly cleared from the mucus layer before they can penetrate to reach the underlying cells and introduce their genetic cargo. Clinical trials testing viral gene vectors (such as adenovirus and adeno-associated viruses) and non-viral vectors (liposomes) for inhaled CF gene therapy have failed to provide clinically significant benefits due to inefficient gene transfer to the target lung cells and generation of therapy-inactivating host immune responses. Furthermore, these gene transfer vectors are immunogenic and elicit undesirable inflammatory responses which can reduce lung function.
Our solution uses inhaled stem cell exosomes to deliver a working copy of the defective CFTR gene, which can efficiently penetrate the protective mucus barrier and efficiently enter the underlying target cells of the airways. These nano-sized stem cell exosomes are regenerative and anti-inflammatory with the potential to reverse disease damage in the lung, and can be tailored via genetic modification of the parent stem cells to create ideal inhaled gene therapy vectors with the potential to treat virtually any respiratory disease have shown 100% mucus penetration and target cell uptake of our surface engineered exosomes in the gold standard in vitro model, which is game changing compared to the state of the art viral vectors which can achieve only 30% at best.
Conclusion of the action: During this Phase 1 Project, OmniSpirant have developed an in-depth understanding of the global market opportunity and assessed the likely criteria demanded by the market, industry, regulatory and healthcare provision systems. The economic viability of the product has been confirmed and a business plan for exploiting the innovation has been constructed. Important partners from industry, academia and the research community have been engaged and some strategic partnerships have been formed. The requirements of these and potential partners have been defined, providing explicit targets for OmniSpirant to achieve. We have also engaged with many investors and reached the final of the European Health Catapult Competition in the Biotech category (to be held in December). A detailed product development plan has been developed and costed, which will fully prepare the product to enter clinical trials.