Finding cures for children's genetic diseases


CMRI announces major international collaboration in gene therapy

In an Australian first, GE Healthcare Life Sciences has teamed up with Children’s Medical Research Institute (CMRI) at Westmead to improve the efficiency and scalability of manufacturing of gene therapy tools to fast-track their accessibility on a global scale.

The partnership would drive the development of new purification technologies for vectors based on adeno-associated virus (AAV). AAV vectors are currently the most promising tools for gene therapy applications, where they are used as microscopic tool (or vehicles) to deliver healthy copies of genes to tissues and cells within patients. 

With more than 800 gene therapies currently in clinical trial, demand for manufacturing technologies is high. AAV vectors are DNA tools used in more than 200 ongoing gene therapy clinical studies. The United States Food and Drug Administration (FDA) estimates that more than 200 such therapies will enter clinical trials annually until 2025. The gene therapy market is projected to reach US$ 5,61 billion by 2027.

The collaboration combines the data from the latest available research on AAV with application testing, advancing a comprehensive understanding of the clinical functionality and the commercial opportunities of AAV-based gene therapies. Children’s Medical Research Institute will share with GE Healthcare Life Sciences AAV variants targeting various, clinically relevant, tissues. GE Healthcare Life Sciences will then design and test ligand prototypes, which Children’s Medical Research Institute will assess. Based on the performance results, GE Healthcare will engineer and commercialize novel improved AAV affinity ligands used to purify AAV vectors.

Olivier Loeillot, General Manager Bioprocess at GE Healthcare Life Sciences says: “The dynamics of the gene therapy space accelerate truly transformative technologies. Our long biomanufacturing expertise combined with Children’s Medical Research Institute’s pioneering research will lead to high-performance purification systems. The industry needs better and more personalized technologies to speed biopharmaceuticals through clinical trials and bring them to market.”

Dr Leszek Lisowski, lead gene therapy scientist at Children’s Medical Research Institute, says: “This is a very rewarding time to be a researcher in gene therapy. Bringing the fruits of our work to the patients requires joint effort between researchers and the industry. The partnership with GE Healthcare Life Sciences will allow us to expedite the development of novel clinical options at a lower cost.”

The AAV affinity ligands resulting from this collaboration will be compatible with GE Healthcare Life Sciences’ resin-based chromatography used in the purification of most FDA-approved biopharmaceuticals.

GE Healthcare Life Sciences helps therapy innovators, researchers and healthcare providers accelerate how precision diagnostics and therapies are invented, made and used. Our products enable biological analysis, research, development and the manufacture of advanced therapies and vaccines. Life Sciences is part of the $19.8 billion healthcare business of GE (NYSE: GE).  With over 100 years of experience in the healthcare industry and more than 50,000 employees globally, GE Healthcare helps efficiently improve outcomes for patients, healthcare providers, researchers, and life sciences companies around the world.