B24. Flexible approach to OGTR licences issued for the delivery of Gene therapy: one licence, multiple therapies

Tracks
Clinical Research
Thursday, June 13, 2024
4:30 PM - 5:30 PM
Parkside 2

Chair & Speakers

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Heidi Mitchell
Ogtr

Chair: Flexible approach to OGTR licences issued for the delivery of Gene therapy: one licence, multiple therapies

Biography

Heidi Mitchell is the Director of the Contained Dealings Evaluation Section at the Office of the Gene Technology Regulator (OGTR). The Section conducts scientific risk assessments and develops risk management plans for clinical trials of GM vaccines or gene therapies and for work with non-plant GMOs both in contained facilities (including maintaining guidelines and certifying contained facilities). Heidi joined the OGTR in 2006 as an evaluator, preparing risk assessments for a range of GMOs. She has also worked in the Regulatory Practice section of the OGTR at the interface between scientific risk assessment and operational regulatory policy. Heidi has been involved in work around the scope of the gene technology legislation. Heidi has a PhD in plant biochemistry and post-graduate research in plant-fungal interactions and on using bacteria other than Agrobacterium for plant transformation. She has published research papers in peer-reviewed journals including Nature.
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Ms Lani Attwood
Assoc. Director Research Operations
Sydney Children's Hospital Network

Flexible approach to OGTR licences issued for the delivery of Gene therapy: one licence, multiple therapies

Biography

Lani Attwood is the Associate Director Research Operations for Kids Research at Sydney Children’s Hospitals Network (SCHN), working to ensure the research infrastructure and support services across SCHN are aligned with the vision of a research-intensive organization. Previously leading strategic and operational initiatives as program manager of the Kids Advanced Therapeutics program since 2020, Lani worked to harness the networks capability and expertise in clinical trials, paediatric care and therapeutic development to establish the networks reputation as a national and global leader in the area of advanced therapeutics. Prior to working for the network, she has held various clinical, research and leadership positions across NSW Health. Lani has been working in health and medical research sector for over 12 years and brings a comprehensive understanding of both the healthcare system and medical research environment in NSW and Australia. Her areas of interest are strategy, research development, governance, clinical trials, regulatory frameworks, advanced therapeutics, research translation and fostering collaboration. Lani’s personal and professional attributes are closely aligned with the networks purpose of helping children and young people live their healthiest lives possible.
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Mrs Geraldine Lester
Assistant Director
Department Of Health, Ogtr

Flexible approach to OGTR licences issued for the delivery of Gene therapy: one licence, multiple therapies.

Abstract

The OGTR was approached with a proposal from the Sydney Children’s Hospitals Network for an application seeking the authorisation of a broad range of clinical trials under one single licence. These trials involved the use of genetically modified Adeno-Associated Viruses (GM AAV) in paediatric gene therapy. Typically, the OGTR issues a licence for trials using a specific GMO (e.g. AAV expressing SMN protein). In this case, a single licence was issued encompassing a broad range of GM AAV-based gene therapies for the treatment of multiple genetic diseases. This session aims to illustrate the perspective from both the Regulator and a clinical site in the preparation and evaluation of flexible risk-based licences.
Perspectives from the site will include discussion on the preparation of the submission and considerations the site had in place prior to submission including the development of standardized process for the handling of GMOs and the training of staff for the conduct of trials.
Perspectives from the OGTR will include the type of information required to conduct a broad assessment, the challenges raised when the specific aspects of the GMO used is unknown, and the specific licence conditions included to address the uncertainty associated with the proposed potential trials.

Biography

Mrs Geraldine Lester is an Assistant Director in the Contained Dealings Evaluation Section at the Office of the Gene Technology Regulator (OGTR). The section conducts scientific risk assessment and prepares risk management plan for work with genetically modified organisms (GMOs) in both OGTR certified facilities and clinical facilities. This includes clinical trials or commercial supply of genetically modified (GM) vaccine/gene therapy and contained work involving GM micro-organisms. Her section also certifies physical containment facilities for work with GMOs. Geraldine joined the OGTR in 2018 as an evaluator and has prepared risk assessments for a range of GMOs.
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