DIR 181

Clinical trial of a genetically modified Herpes virus for the treatment of cystic fibrosis

Licence details

Licence number:
DIR 181
Licence status:
Limited and controlled

Novotech (Australia) Pty Limited

Parent organism:

Herpes simplex virus-1

Modified trait:

Human therapeutic – replication incompetent; Human therapeutic – protein expression

Licence issue date:

Novotech (Australia) Pty Limited is conducting a clinical trial of genetically modified (GM) Herpes simplex virus-1 as a treatment for cystic fibrosis.

Cystic fibrosis is an inherited disease caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The GMO is a gene therapy product intended to repair this genetic defect by restoring a functional copy of the CFTR gene to lung cells.

It will be manufactured overseas and imported into Australia.

The RARMP concluded that the clinical trial poses low to moderate risks to people and negligible risks to the environment. To manage these risks, licence conditions have been imposed requiring that trial participants are:

  • seronegative for HSV-1
  • monitored over the course of the trial for primary HSV-1 infection
  • offered anti-viral medication if they acquire an infection.

Up to 15 people with cystic fibrosis are permitted to be treated with the GMO in a hospital setting, over a 3 year period.

Effective protection of clinical trial staff from aerosolised GMO is also required.

A range of additional controls are imposed to minimise the potential for exposure of people other than trial participants, and susceptible animals, to the GMO.

Date application received:
RARMP open for public consultation:
RARMP closed for public consultation:


Licence decision - notification

Outlines the Regulator’s decision to issue a licence following the assessment of this application.

Licence decision - Q&A

FAQs on the licence application and the Regulator’s decision to issue a licence for this application.

Risk assessment and risk management plan - summary

A summary of the Risk Assessment and Risk Management Plan prepared as part of the assessment of this application. It provides a brief description of the licence application, the risk assessment and risk management plan.

Risk assessment and risk management plan - full version

The final risk assessment and management plan prepared to support the Regulator's decision. It describes the GMO(s) and proposed work with the GMO(s) and provides an assessment of potential risks posed by the GMO(s). It also includes a summary of submissions received during the public consultation process.

Licence conditions

Sets out the licence conditions imposed by the Regulator, including the licence holder’s general and specific obligations and reporting requirements.

Last updated: