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Faster, cheaper way to make gene-therapies wins grant

8 August 2018
Funding to progress new gene delivery method
The NSW government has awarded $500,000 to Indee Labs to partner with the University of Sydney to eliminate the need for viruses to make gene-modified cells capable of curing some forms of blood cancer.
Gene therapy is changing the face of cancer therapies

Gene-modified cells are changing the face of cancer therapies

The use of viruses is a major roadblock in progressing cell therapies as a cancer cure because it increases the cost and complexity of producing the treatments. Simplification using the method proposed by Indee could make the whole process quicker, cheaper and more widely accessible.

Announced today by NSW Health Minister Brad Hazzard MP, the NSW Medical Device funding means the partners can progress a technology allowing cells to be altered or ‘transfected’ at scale for use in gene therapies. 

Current gene therapies are too slow and expensive to make, and manufacturing isn’t scalable to meet even a small fraction of global demand
Indee Labs Australia chief executive, Dr Warren McKenzie

“The problem we are addressing is that current gene therapies are too slow and expensive to make, and manufacturing isn’t scalable to meet even a small fraction of global demand,” says Indee Labs Australia chief executive, Dr Warren McKenzie.

Instead of using viruses to transport genetic material into cells, the company employs a device (a microfluidic vortex shedding assembly) to disrupt a cell membrane, allowing genetic material such as mRNA into the cell

It has already demonstrated this with human blood cells and T cells, and lab tests show it is faster, safer and more scalable than competing technologies, such as viruses or ‘electroporation’, which involves delivering an electric shock to a cell to punch a hole in its membrane.

The partners will use the funds to compare Indee Labs’ technology with the electroporation gene delivery method for future use in clinical trials led by Dr David Gottlieb, Professor in Medicine at the University of Sydney and Westmead Institute for Medical Research.

We are very optimistic the Indee method will allow us to rapidly roll out locally manufactured gene modified T cells to Australian patients with blood cancers
Dr David Gottlieb, Professor in Medicine, University of Sydney

“We are very optimistic the Indee method will allow us to rapidly roll out locally manufactured gene modified T cells to Australian patients with blood cancers,” said Professor Gottlieb. “If that is successful, the method will put us in a great position to apply the technology to a wide group of cancer sufferers.”

Gene therapy breakthroughs

The field of gene therapy has made world-shattering discoveries that will eventually cure many genetic disorders and forms of cancer, but these therapies are astronomically expensive.

For example, the first gene therapy approved by the US Food and Drug Administration (Kymriah, marketed by Novartis), is spectacularly effective against a rare form of leukaemia, causing remissions in 83 percent of patients under 25 when conventional treatments have failed. But the price tag is AUD $640,000.

Yescarta, another recent FDA-approved gene therapy, genetically reboots a patient’s immune cells to kill aggressive forms of a blood cancer, non-Hodgkin’s lymphoma. The cost will be AUD $504,000.

“Hundreds of clinical trials are underway around the world to develop gene therapies, most of which rely on a gene-delivery step in their manufacture,” says Dr McKenzie. “We estimate that if these are successful, around half of all cancer deaths will be addressable.”

While the use of microscopic turbulence will reduce the cost of delivering genes to modify cells, its real value is to create a dramatically simpler process for creating new gene therapies.

Most gene therapies under development today rely on engineered viruses to deliver genetic material into a cell.

These viral ‘vectors’ can be injected intravenously or delivered directly into a specific tissue in the body, where they are taken up by individual cells. Alternately, a sample of the patient's cells can be removed and exposed to the vector in a lab setting.

But virus-delivered genetic material can come with problems. For example, surface-presented viral antigens increase the risk of immunogenicity that provokes an autoimmune response in the body. The risk of harmful mutations means additional quality control procedures are required. And the US FDA, for example, requires patients to be tracked for up to 15 years after infusion.

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