New facility in Western Sydney to deliver life-saving medical technology

Children with genetic diseases and cancers will be given faster access to life-saving gene and cell therapies through a new commercial scale viral vector manufacturing facility in Western Sydney.

The NSW government has called on industry and investors to submit expressions of interest to support the growth and operations of the facility, to be built in the Westmead Health and Innovation District.

Premier Dominic Perrottet says the facility will manufacture high-quality clinical grade viral vectors and will serve the Australian, broader Asia-Pacific and global markets for research and clinical trials.

Based at The Children’s Hospital at Westmead (CHW), Sydney Children’s Hospitals Network (SCHN) is home to one of the only viral vector manufacturing facilities in Australia, after the $25 million start-up investment was announced in December 2019. This followed seed funding from the NSW Office of Health and Medical Research (OHMR) in partnership with Luminesce Alliance to establish a pilot viral vector manufacturing facility.

Viral vectors are modified viruses used as a vehicle to safely deliver vaccines and gene therapies into the cells of a patient with a genetic condition. They are key components of gene therapies for rare genetic diseases and cell therapies for cancer.

Greater access to these therapies brings new hope to families across Australia. Sydney woman Gemma Najem says expanding local production of viral vectors is crucial, after seeing how gene therapy changed her son’s life.

Her son Jonathan was diagnosed with Spinal Muscular Atrophy (SMA) as a newborn. SMA is a rare genetic disorder causing progressive muscle weakness. Babies with SMA struggle to meet developmental milestones, including holding their head up, sitting up by themselves, standing and walking. In Australia, one in 10,000 births is affected by SMA and it’s the leading cause of death in babies under two years of age.

Babies confirmed with SMA and treated with gene therapy soon after birth can achieve age-appropriate motor milestones. However, babies with SMA need to receive the treatment within their first few weeks of life and often parents are in the dark about the condition until the irreversible damage begins.

After diagnosis through newborn-screening, Jonathan received access to gene therapy at 4-and-a-half months. Gemma says viral vector technology has changed his day-to-day life and given him the best chance at mobility, strength and health.

“For the families of children with rare genetic conditions, local production of viral vectors makes massive difference because it can fast track life-saving and life-changing treatments,” Gemma said.

One of the world’s leading gene therapy experts, Professor Ian Alexander, supported the family throughout, assisting with Jonathan’s quality of care. Prof Alexander who is Head of the Gene Therapy Unit, a joint initiative of CMRI and CHW, is also part of the viral vector manufacturing facility project.

“You could say the most elegant way of fixing a faulty gene is like fixing a spelling error in a document, with that sort of precision. That’s the journey we’re on and we could only dream it was possible five years ago. The capacity to repair faulty genes at that level is now with us,“ Prof Alexander said.

The new commercial scale facility will have space for manufacturing equipment with 500L production capacity, laboratories and workspace. It’s set to be operational in 2023.