New gene therapy to target airway and lungs via nasal spray

Scientists at Mass General Brigham have engineered a groundbreaking gene therapy delivery method using a nasal spray called AAV.CPP.16. The innovative approach allows precise targeting of lung and airway cells, showing remarkable performance in preclinical studies across mouse and primate models. Researchers successfully demonstrated potential applications in treating pulmonary fibrosis and preventing SARS-CoV-2 virus replication. This breakthrough represents a significant advancement in respiratory gene therapy with promising translational potential for future medical treatments.

"We noticed that AAV.CPP.16... efficiently targeted lung cells" - FengFeng Bei, Brigham and Women's Hospital

US researchers have engineered a novel gene therapy to target the airway and lungs via a nasal spray.

Key Points

1 Novel nasal spray gene therapy targets airway and lung cells effectively

2 Outperforms previous AAV delivery methods in multiple models

3 Demonstrates potential for pulmonary fibrosis and viral infection treatments

4 Developed by Mass General Brigham researchers

For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body. It is commonly done by using adeno-associated viruses (AAV) gene therapy.

To improve the AAV's ability to deliver therapeutics specifically to the lungs and airway, researchers at the Mass General Brigham engineered a new version, called AAV.CPP.16, which can be administered with a nasal spray.

In preclinical models, AAV.CPP.16 outperformed previous versions by more effectively targeting the airway and lungs and showing promise for respiratory and lung gene therapy, said the researchers in the paper published in the journal Cell Reports Medicine.

"We noticed that AAV.CPP.16, which we initially engineered to enter the central nervous system, also efficiently targeted lung cells," said senior author FengFeng Bei, from the Department of Neurosurgery at Brigham and Women's Hospital.

"This prompted us to further investigate AAV.CPP.16 for intranasal gene delivery to the respiratory airways," Bei added.

In the study, AAV.CPP.16 outperformed previous versions (AAV6 and AAV9) in cell culture, mouse models, and non-human primate models.

“Our findings highlight AAV.CPP.16 as a promising vector for respiratory and lung gene therapy,” the team said.

They then used the more efficient tool to deliver scar-preventing gene therapy for pulmonary fibrosis, using a mouse model of the respiratory disease.

They also used the tool to deliver gene therapy for a viral infection, where the therapy prevented the replication of the SARS-CoV-2 virus in a mouse model of Covid-19.

"Although further research is needed, our findings suggest that intranasal AAV.CPP.16 has strong translational potential as a promising delivery tool for targeting the airway and lung," said Bei.

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