What gene therapy is, and where it stands in Australia
Healthdirect Australia describes gene therapy as a type of medical treatment that aims to prevent or treat genetic conditions, by delivering a corrected gene into a person’s cells to correct a mutation. It also says gene therapy is mostly still in research and clinical trials, and is not generally available in Australia.
General information, not medical advice. Your own doctor is the person to ask about any treatment. Healthdirect’s guide to genetic disorders and the OGTR’s page on GMOs are the official places to read more.
- Described by
- Healthdirect, the national public health information service
- Products regulated by
- The TGA
- GMOs regulated by
- The Gene Technology Regulator
- Where it mostly is
- Research and clinical trials
Genes, in one paragraph
Healthdirect explains that genes carry the instructions that tell the body how to work, and that they are found in every cell. Genes are made of DNA, and a random change in DNA is called a mutation. Most mutations have no noticeable effect, but sometimes a change alters how a gene works, which can lead to a genetic problem or disorder.
Healthdirect gives two diseases as examples of where gene therapy has already been used as a treatment: haemophilia and spinal muscular atrophy (SMA). It does not say gene therapy is available for either here; its own summary is that the field is mostly still in research and trials.
Gene technology, the wider family
The Office of the Gene Technology Regulator (OGTR) describes gene technology, also called genetic engineering or genetic modification, as ways to make changes to genes: modifying or removing a gene directly, or moving a gene from one species to another.
An organism changed this way is a genetically modified organism, or GMO, and so is an organism that has inherited modified traits from one. Among the uses of GMOs, the OGTR lists medicine: developing new ways to diagnose and treat disease. It also notes that genetic modification is widely used to make medicines such as insulin. Those two uses are not regulated the same way, and the difference matters for what “gene therapy” means.
Three things that involve genetic modification
The TGA’s guidance puts products that involve GMOs into different groups, each with its own rules. This table sets out three, as the TGA and the OGTR describe them.
| What it is | Examples the regulator gives | How it is treated |
|---|---|---|
| A medicine made with the help of a GMO | Vaccines, monoclonal antibodies, and hormones such as insulin and growth hormone | The TGA calls these biological medicines and regulates them as registered prescription medicines. A trial of a product made from a GMO, with no viable GMO in it, needs no authorisation from the Gene Technology Regulator. |
| A GMO used as the medicine itself | Live attenuated vaccines, viral vectors and modified somatic cells | Covered by the TGA’s guidance for prescription medicines that contain GMOs, which tells sponsors to consult the OGTR, including for use in a clinical trial. |
| Human cells modified to carry out a function they did not have when collected | Genetic modification of cells, such as CAR T cells and induced pluripotent stem cells (iPSCs) | Regulated as biologicals, in Class 4, the TGA’s high-risk class. The TGA’s guidance on GMO medicines does not apply to cell and tissue therapies that are defined as biologicals. |
Each row is worded from the regulator’s own guidance. Which rules apply to one particular product is set out in the TGA’s guidance, covered on rung 02, how the TGA regulates it.
Why a viral vector counts as a GMO
The OGTR’s guidance on clinical trials explains that the Gene Technology Act defines a GMO as an organism modified by gene technology, and an organism as any biological entity that is viable, capable of reproduction or capable of transferring genetic material. The OGTR reads that broadly enough to include replication-defective viral vectors, and nucleic acids that can give rise to infectious agents once inside a host cell.
That is why putting a GMO into a person, in a trial, needs a licence from the Gene Technology Regulator, unless it is a modified human somatic cell that meets specific conditions. Rung 05, the Gene Technology Regulator, walks through those conditions and the two kinds of licence.
Gene editing
Whether an organism changed by gene editing or RNA interference is a GMO depends on the technique used, and the Regulator publishes an overview of how it treats organisms modified with gene editing and other new technologies.
Embryo research and mitochondrial donation
The NHMRC explains that the Research Involving Human Embryos Act 2002, together with the Prohibition of Human Cloning for Reproduction Act 2002, prohibit human cloning for reproductive purposes and regulate research that uses human embryos, with strong penalties for breaking them.
The NHMRC also explains that Maeve’s Law, which took effect in October 2022, allowed mitochondrial donation in research, training and reproduction, and that its Embryo Research Licensing Committee is the licensing authority. The NHMRC describes mitochondrial donation as a technique that “could help prevent certain rare mitochondrial diseases”.
Where the rules go next
Three sets of rules come up again and again in this guide: the TGA’s rules for the product (rung 02), the Gene Technology Regulator’s rules for the GMO (rung 05), and ethics review of the research (rung 04). Because healthdirect places most gene therapy in research and trials, rung 03 on clinical trials is where many readers will want to go next.