Innovative Nasal Spray Gene Therapy Targets Airways and Lungs

Researchers at Mass General Brigham have developed a novel gene therapy that can be administered via nasal spray to specifically target the respiratory airways and lungs. This breakthrough involves a new adeno-associated virus (AAV) variant called AAV.CPP.16, which enhances delivery efficiency to lung tissues. Traditionally, successful gene therapy depends on effectively delivering therapeutic molecules to the correct locations, often using viral vectors such as AAV.

In preclinical studies, AAV.CPP.16 demonstrated superior targeting capabilities compared to earlier versions like AAV6 and AAV9. These studies included cell cultures, mouse models, and non-human primates. The team successfully used this vector to deliver gene therapy aimed at preventing pulmonary fibrosis, a serious lung condition, as well as to combat viral infections such as COVID-19 by inhibiting the replication of SARS-CoV-2 in mice.

Remarkably, Dr. FengFeng Bei, senior author from Brigham and Women's Hospital, explained that AAV.CPP.16 was initially engineered for central nervous system applications but was found to effectively target lung cells, prompting further investigation for respiratory therapies. These findings suggest significant translational potential, offering a promising new avenue for treating lung diseases through minimally invasive intranasal administration.

The research, published in Cell Reports Medicine, highlights the potential of AAV.CPP.16 to serve as a powerful delivery tool in gene therapy, opening doors to targeted treatments for respiratory conditions and viral infections. Further studies are needed, but this represents a substantial step forward in respiratory gene therapy technology.