Gene therapy researchers at Children's Medical Research Institute (CMRI) are developing a new way to target the lungs more effectively, with the aim of improving treatment of cystic fibrosis.
Associate Professor Leszek Lisowski and researchers Dr Andrea Perez-Iturralde, Maddy Knight, and Kim Dilworth were excited to show their progress during a recent lab tour with the Cure4 Cystic Fibrosis Foundation and the Team Simon Foundation, who have generously supported this research.
It’s rare for us to have our researchers in the same room as patients, parents, clinicians and the foundations funding our work. But on this special occasion we were joined by Olivia, an ambassador for Cure4CF Foundation who is living with CF, as well as Harry and Teresa Bazouni, co-founders of the Team Simon Foundation whose son Simon has CF, as well as Professor Hiran Selvadurai from the Children’s Hospital at Westmead who is collaborating with A/Prof Lisowski on this project. All were excited to be in attendance to hear about CMRI’s progress toward a gene therapy for cystic fibrosis patients.
“I had a fantastic time connecting with our research partners at CMRI,” said Cure4CF CEO, Suzy Dimaline. “A/Prof Lisowski and his team are making significant strides in addressing cystic fibrosis at its core. Touring their labs allowed me to witness their important work first hand.”
Andrea explained that one of their key aims is “the development of a vector that is able to reach the lungs, because there is no specific vector that goes to the lung.” Vectors are microscopic delivery vehicles that can carry a healthy copy of a gene to replace a malfunctioning gene, an approach already successfully used to cure other genetic diseases.
The team explained the complex process of designing new vectors and were excited to share they had narrowed the search down from an initial 64 million vector options, to a small subset that appears to successfully target the lungs.
Maddy explained: “I found these peptides," which act as the address label on the vectors, "that had been previously published. I inserted them into these three promising vectors, making a total of 18 new variations. Of those 18, there was one peptide that was more successful at reaching the lung by about 7-20%. But more interesting is that this vector, which tends to go towards the liver, was significantly de-targeted from the liver which is very exciting.”
“This is very early, and we need to do many other experiments, but it is something that is already very exciting. Now we try to build on this success to make the vectors even better.”
Everyone had the opportunity to witness Maddy's discovery in the lab, seeing tissue culture cells that were targeted with one of the new vectors, which turned them green with fluorescent reporter protein!
CMRI received the major Holckner Family CF Impact Grant from Cure4 Cystic Fibrosis Foundation in 2023. It is the second major grant to CMRI researchers from Cure4 Cystic Fibrosis Foundation, thanks to fundraising efforts of the Team Simon Foundation who have a long history with CMRI.
"It was an absolute pleasure meeting the team at CMRI alongside our partners from CURE4CF,” said Teresa Bazouni. “This opportunity wouldn't have been possible without the support from our generous sponsors and donors. We’re grateful to be a part of life-changing moments."
“It’s inspiring to realise that this is where transformative advancements take place,” said Suzy. "I feel honoured to be in the company of such talented individuals dedicated to creating a future free from cystic fibrosis.”