International biotech partnership brings gene therapy closer to the clinic

Top gene therapy researchers Professor Ian Alexander and Dr Leszek Lisowski will lead a new research program in genetic engineering that is set to fast-track gene therapy treatments for children, thanks to an international partnership between the Children’s Medical Research Institute (CMRI) and LogicBio Therapeutics, Boston.

The deal will see the partners develop new viral vectors, which transport a healthy copy of a gene into a patient’s cells to either replace or edit the faulty gene.

Professor Alexander, head of the Gene Therapy Research Unit, a joint venture of CMRI and the Sydney Children’s Hospitals Network (SCHN), and Dr Lisowski, leader of the Translational Vectorology Group at CMRI, will develop the next-generation adeno-associated viral (AAV) vectors to improve those currently in use, many of which were discovered more than ten years ago.

“We are confident that we can further improve on the performance of current AAV vectors, expanding their utility in a range of tissues, while also improving manufacturability and reducing cost,” said Dr. Lisowski.

"We’re optimistic that we can significantly improve the performance of AAV vectors through this collaboration,” said Fred Chereau, CEO of LogicBio. “These next-generation vectors will strengthen our platform and expand our pipeline. We also expect them to bolster other important approaches to genetic medicine, including gene therapies.”

Genetic medicine has the potential to dramatically change the lives of children with rare genetic diseases, like three-year-old Charlize Gravina.

Charlize and her twin brother Isaac were born with propionic acidemia, a serious metabolic disorder. Isaac required a liver transplant when he was just two years old.  Sadly, due to post-surgery complications, he died a short time after the transplant.

Within months, his family went through the heart-breaking decision of submitting Charlize to the same procedure. She has now had two liver transplants and her parents, Julie and Paul, are so excited to hear about this collaboration.

“When you are told there is no treatment, no cure,’’ Mrs Gravina said, “all you want to do is change things for other people, so they don’t have to go through what we’ve gone through.’’

The Gravinas are now focused on providing the best future they can for Charlize and her big sister, Amelia, who wants to be a medical researcher – but they also want to help future generations.

Professor Alexander, who is a scientist and a clinician, said they hoped to produce significant outcomes for patients like the Gravinas. They are even using a sample from Charlize’s liver in their research.

“We’re trying to get to a point where instead of a liver transplant in a very young infant, we can genetically repair the liver without major surgery. It’s a very exciting time to be doing gene therapy.’’

SCHN and CMRI have long contributed to pioneering work in the clinical translation of cell and gene therapies, including Prof Alexander’s team being the first in Australia to treat a genetic disease (SCID-X1) by gene therapy.

He said the investment from LogicBio was also a good sign for the local industry, acknowledging and supporting the local expertise.

“ It recognises that we are one of the key groups in the world doing this work. I think this is an indication of the growing recognition of the strength of Australian researchers in this highly specialized field, contributing to the globally exciting development of gene therapies. It is further growth of the Australian biotech industry and will have a knock-on effect. We see it as the beginning of something much greater. It is about moving technology into the clinic which, in future, will benefit many more patients by offering new and better treatment opportunities.’’

This latest announcement follows on from the news earlier in the year that NSW Health will provide funding to establish capacity to manufacture gene transfer vectors at Westmead through Paediatrio. This project is also run jointly by Professor Alexander and Dr Lisowski at CMRI and the SCHN.