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Australia: Therapeutic / Stem Cell

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Highly Regulated

Somatic cell gene therapy clinical trials permitted.

Gene therapy is permitted but must comply with clinical trial regulations. Amended regulations implemented in 2019 make it quicker and easier for medical researchers to get approval for gene therapy research and to progress to clinical trials with patients. Gene editing techniques that pose no different risks, and cannot be distinguished from conventional methods, do not require additional regulation.

Gene therapy must be approved through the Therapeutic Goods Administration (TGA), which oversees all clinical trials of medicines. The TGA is guided by the Gene Technology Act 2000, which is a plan for the regulation of GMOs. The act includes the Gene Technology Regulations 2001, which require a license from the Gene Technology Regulator before introducing a genetically engineered organism into a human, including human clinical gene therapy trials.

Clinical trials can be approved in two ways: through a Clinical Trial Notification (CTN) or Clinical Trial Exemption (CTX) scheme. Under the CTN, a Human Research Ethics Committee must first review the trial and then notify the TGA. Under the CTX, the TGA directly reviews the trial, which may not proceed until approval is granted. CTX schemes are typically used for clinical trials involving new technology and new treatment concepts.

Products/Research

  • Bubble-boy disease (SCID-X1): The Gene Therapy Research Unit at Children’s Medical Research Institute and The Children’s Hospital at Westmead was the first group to treat a genetic disease in Australia.
  • Inherited liver disease (OTC-deficiency): The Gene Therapy Research Unit is preparing for human clinical trials of an AAV-based treatment, which is considered safer than a liver transplant.
  • Ovarian and gastric cancers: A partnership between the Melbourne company Cartherics, Hudson Institute of Medical Research, Monash University, Mesoblast and Cell Therapies developed a CAR T-cell therapy that began clinical trials in 2019.
  • B-cell Acute Lymphoblastic Leukemia (ALL): Researchers at Westmead Institute for Medical Research conducted a clinical trial using CAR T-cell immunotherapy to prevent relapse after treatment.
  • Leukemia and lymphoma: Researchers at WIMR and Westmead Hospital approved to conduct a clinical trial for CAR T-cell immunotherapy.
  • Cancer: Research conducted in multiple areas, including multiple myeloma, colorectal cancer and using gene editing to study genes that suppress tumors.

Regulatory Timeline

2019: 2019 Amendments to the Gene Technology Regulations 2001 that ease restrictions on gene therapy commence.

2019: Gene Technology Regulator conducts technical review of Gene Technology Regulations 2001 to clarify the regulatory status of organisms developed using NBTs.

2017: The Therapeutic Goods Administration (TGA) announces reforms that tighten regulations of CAR T-cell immunotherapy.

2001: Gene Technology Act 2000, a plan for the regulation of GMOs in Australia, defines gene technology as any technique for the modification of genes or other genetic material.

1991: GM therapeutic goods (including clinical trials) regulated under the Therapeutic Goods Act 1989.

NGO Reaction

None

Additional Resources

Click on a country (eg. Brazil, US) or region (eg. European Union) below to find which human / health products and processes are approved or in development and their regulatory status.

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Human / Health Gene Editing Index
Compare Regulatory Restrictions Country-to-Country

Gene editing regulations worldwide are evolving. The Gene Editing Index ratings below represent the current status of gene editing regulations and will be updated as new regulations are passed.

Colors and ratings guide
 

Regulation StatusRating
Determined: No Unique Regulations*10
Lightly Regulated8
Proposed: No Unique Regulations†6
Ongoing Research, Regulations In Development5
Highly Regulated4
Mostly Prohibited2
Limited Research, No Clear Regulations1
Prohibited0
Lightly Regulated: Gene and stem cell therapies regulated with minimal restrictions and requirements.
*Determined: No Unique Regulations: Gene and stem cell therapies regulated as phamaceuticals with no additional restrictions.

†Proposed: No Unique Regulations: Decrees under consideration for gene and stem cell therapies that would not require unique regulations beyond current restrictions on pharmaceuticals.

Therapeutic:
Gene editing of adult human cells, including gene therapy and stem cell therapy, that is used to treat and cure disease. Recent breakthroughs include CAR T-cell therapy, which uses patients’ own immune cells to treat their cancer.
Germline:
Gene editing of the human embryo or germline that results in genetic changes that are passed down to the next generation. This type of gene editing is the most controversial because changes are inherited and because it could theoretically be used to create “designer babies”. A Chinese scientist announced in 2018 that he had successfully edited twins that were brought to term. International backlash from the announcement has resulted in China and other countries working to clarify regulations on germline gene editing.

Rating by Country / Region
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Country / RegionTherapeuticGermlineHuman Rating
Japan888
Brazil402
Canada402
Russia1057.5
Argentina513
Israel825
Australia402
China846
US402
Chile412.5
New Zealand402
Ukraine1057.5
Central America111
Paraguay111
Uruguay111
India402
UK444
Mexico804
EU402
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Gene editing is a set of techniques that can be used to precisely modify the DNA of almost any organism. It is being used for applications in human health, gene drives and agriculture. There are numerous gene-editing tools besides CRISPR-Cas 9, which gets most of the attention because it is a comparatively easy tool to use.

Gene editing does not usually involve transgenics – moving ‘foreign’ genes between species. It also refers to a specific technique in contrast to the general term GMO, which is scientifically ambiguous, as genetic modification is a process not a product. Most gene editing involves creating new products by deleting very small segments of DNA (sometimes in agriculture called Site-Directed Nuclease 1 or SDN-1 techniques), which can silence a gene or change a gene’s activity. Countries are evaluating whether or not to regulate this type of gene editing, since it is so similar to natural mutations. The GLP’s Gene Editing Index ratings reflect the regulatory status of SDN-1 techniques, which are the most liberally regulated and will generate most products in the near term.

To develop different products, gene editing can change larger segments of DNA or add DNA from other species (a form of transgenics sometimes in agriculture called SDN-2 or SDN-3 techniques). While many countries are not regulating or lightly regulating SDN-1 techniques, most are moving toward tightly regulating or even restricting SDN-2 and SDN-3.

For more background on the various gene editing SDN techniques, read background articles here and here.

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