Loxegen's nanoparticle gene therapy for cystic fibrosis (CF) has secured a significant $1.5 million grant from the Australian Government’s $230m Medical Research Future Fund (MRFF), with additional contributions pushing the total funding to over $3 million.
This funding boost marks a significant step forward in the pursuit of more effective treatments for CF.
This project involves the collaborative efforts of several academic institutions, research organisations, and consumer groups like Cystic Fibrosis Community Care (CFCC). Led by Andrew Venables, CEO of Loxegen and father to a child living with CF, this partnership aims to revolutionise treatment for all people with CF, regardless of their CFTR mutations.
Loxegen’s nanoparticle gene therapy aims to deliver a functional copy of the CFTR gene directly into a patient’s lung cells, via inhalation. By introducing a functional copy of the CFTR gene, this therapy aims to restore CFTR channel function in the lungs.
Gene therapy differs greatly from the current treatments available for CF. CFTR modulators, like Trikafta, aim to help defective CFTR proteins fold correctly and stay open at the cell surface. Gene therapies, however, aim to treat CF at the root cause, by delivering a functional CFTR gene that can then make correct CFTR proteins. In this way, gene therapies have the potential to help all people with CF, regardless of their gene mutation.
This project has been supported by an initial grant from CFCC in 2016 of $149,000 which contributed to the preliminary data used in the MRFF application.
The MRFF funding will support crucial research activities to further develop this technology, including optimising and validating the nanoparticle gene therapy for targeted delivery into lung cells. Utilising CF-induced stem cells and animal models, researchers aim to understand how the therapy interacts and moves within cells and throughout the lungs.
Gene therapy research has evolved since the CFTR gene was discovered in 1989 but remains a complex research area. This significant investment is a step in the right direction, and we will monitor its journey closely over the coming years.
CFCC's involvement as a consumer partner in this grant underscores the importance of community input in shaping the future of CF treatment. We look forward to providing further updates on the progress of this research project.
Read Loxegen’s media release here.
Image from: Loxegen