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Respirable Advanced Therapeutics for Cystic Fibrosis & other Lung Diseases

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A new way of treating cystic fibrosis

Using gene therapy to replace mutated genes with a healthy copy to help cystic fibrosis patients is not a new concept, but the mucus barrier has proved an obstacle. An innovative approach may have found a way to penetrate the protective mucus barrier and efficiently enter into the underlying target cells of the airways.

Health

Cystic fibrosis (CF) is a genetic disease that causes persistent lung infections and, over time, limits a person’s ability to breathe. According to the Cystic Fibrosis Foundation, over 80 000 people are infected with the disease. As there is no cure, those living with CF must manage their symptoms through airway clearance, inhaled medicines, individualised fitness plans, and enzyme supplements. Now, thanks to early stage development of regenerative gene therapies, CF patients may, in time, have a new method for treating their symptoms. At the forefront of this effort is OmniSpirant Limited, an Irish company dedicated to developing a first-in-class regenerative gene therapy for CF. Thanks to the support of the EU-funded ReSpire project, the company is one step closer to transforming respiratory medicine. For the first time, OmniSpirant has demonstrated the widespread and efficient entry into the high number of cells required for truly successful CF gene therapy. “OmniSpirant has been developing inhaled bioengineered exosome therapeutics that can be delivered via a tailored aerosol spray,” explains Gerry McCauley, OmniSpirant CEO and ReSpire project coordinator. “With the support of EU funding, we were able to thoroughly plan our business strategy and product development so this new approach may eventually offer an effective solution to the entire CF population.”

A more effective, safer therapy

The aim of CF gene therapy is to replace the mutated CFTR gene with a healthy copy. Although the concept was proven over 20 years ago, to date it has failed to show significant clinical benefits. “The main problem is efficiently delivering the gene to lung airway epithelial cells,” says McCauley. “Specifically, the thick mucus in the lungs of CF patients traps the carriers used to deliver inhaled CFTR gene therapies like nanoparticles and viral vectors.” To overcome this mucus barrier, the OmniSpirant platform uses surface engineered stem cell exosomes as regenerative carriers for inhaled gene therapy. Exosomes are nanosized vesicles that are naturally produced by virtually all cells and are involved in cell-to-cell communication. “Our solution uses inhaled stem cell exosomes that have been surface engineered to deliver a working copy of the defective CFTR gene, which can efficiently penetrate the protective mucus barrier and efficiently enter into the underlying target cells of the airways,” explains McCauley. According to McCauley, these stem cell exosomes are anti-inflammatory and non-immunogenic, and can be tailored via genetic modification of the parent stem cells to create ideal inhaled gene therapy vectors for virtually any lung disease. Furthermore, the nanosized stem cell exosomes have regenerative properties and may be able to reverse disease damage in the lung. “The hope is that these exosomes will be more effective, safer, and applicable to 100 % of the global CF population,” McCauley says.

A range of different uses

The enhanced delivery properties of the platform also have the potential to maximise the regenerative effects mediated by the stem cell exosomes. “In the future, it is hoped that this platform will prove useful for treating not only CF, but also chronic obstructive pulmonary disease, lung cancer, and many other respiratory diseases,” adds McCauley.

Keywords

ReSpire, OmniSpirant, cystic fibrosis, Cystic Fibrosis Foundation, regenerative gene therapies, respiratory medicine

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