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
- Genetic Literacy Project’s FAQ on gene editing
