Australian-first facility to advance treatment for genetic diseases

Australian-first facility to advance treatment for genetic diseases

Twelve-year-old Jude with Health Minister, Ryan Park.

A new viral vector manufacturing facility being built in the Westmead precinct is set to change the treatment landscape for patients with genetic diseases, cancers and viral infections, enabling gene therapies to be trialled and delivered sooner.

Viral vectors are a key component of many cell and gene therapies. The technology uses viruses that are harmless to humans to carry genetic therapies into the body and target organs and systems that cause significant human diseases.

The NSW Government has committed $134.5 million to establish the Facility and manufacture viral vector products for research and clinical trials, and have now announced the establishment of a new company to operate the world-leading facility.

Viral vector manufacturing announcement

“This is cutting-edge, world class work happening right here in Western Sydney," Daniel Mookhey, NSW Treasurer, said.

“This commercial-scale facility will allow us to meet local manufacturing demand, and further establish us as a respected leader in this field."

The new facility is hoped to help address the global bottleneck in viral vector manufacturing.

“These therapies are expanding rapidly, targeting more and more genetic diseases, cancer and infections – significantly increasing the number of patients who can and will benefit from viral vectors," Ryan Park, NSW Health Minister said.

“This expansion in potential applications, treatments and patient benefits is not however matched by current manufacturing capacity, either locally or globally, which is causing a delay in us achieving these benefits.”

For families like Megan Maack's, the facility offers renewed hope. Megan's children, Isla (14) and Jude (12) live with Sanfilippo Syndrome, a rare genetic condition causing childhood dementia. Unfortunately, with viral vector manufacturing capability not previously available in Australia, Isla and Jude weren't able to receive gene therapy in time for it to make a difference but Megan is optimistic about the change to come. 

NSW Health Minister meets with Megan Maack, and son, Jude.

"I've worked ever since my children were diagnosed to accelerate therapies for their condition, and more broadly for other children with dementia so I am extremely happy to see the progress, although bittersweet," Ms. Maack said.

"I think within the next decade we're going to see an avalanche of new treatments so there is hope. It's no longer the hopeless landscape I walked into 11 years ago."

Professor Ian Alexander, head of the Gene Therapy Research Unit at Sydney Children's Hospitals Network and the Children's Medical Research Institute, said the facility would have worldwide impact.

Professor Ian Alexander discusses impact of gene therapy

"Right now, there are 3,000 genetic therapies in the developmental pathway globally, many in Australia, but most of them are in a laboratory. What this initiative is going to help us do is take those therapies out of the laboratory and into the clinic at speed and with safety," Professor Alexander said.

"The overall aim is to bring the best possible therapies to people at the earliest possible moment, children and adults alike. We intend for these therapies to not only be available to patients around Australia, but around the world so it really is global impact starting here in New South Wales," Professor Alexander said.

"We are at a profoundly exciting moment in medical history and are really starting to see the therapeutic impact of the genomic revolution."

Stage 1 of the facility is already operational for use in gene therapy research with the first clinical trial anticipated to commence in 2025 using vectors from the facility.

The Stage 2 facility is currently under construction and once completed will provide NSW with a commercial-scale viral vector manufacturing facility capable of meeting the growing demand for viral vectors.

Completion of construction works is expected by the end of 2024, with Good Manufacturing Practice certification to follow soon after. The company is to be registered as Viral Vector Manufacturing Facility Pty Ltd. It will manufacture the vectors and supply them to specialised healthcare staff, who will deliver the therapies to patients.