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Cell and Molecular Therapies Research

The Department of Cell and Molecular Therapies (CMT) is located at one of Australia's oldest tertiary referral public healthcare institutions with an outstanding record in clinical research and early adoption of novel technologies.

We are adjacent to Australia's first tertiary education institution, the University of Sydney, ranked in the top 100 universities in the world.

Led by Professor John Rasko AO, a clinical haematologist, pathologist, and scientist with an internationally renowned track record in gene and stem cell therapy, experimental haematology and molecular biology.

Our Firsts in Cell and Gene Therapy

  • FIRST clinical gene therapy trial of AAV in Australia – Gene delivery of Factor IX for haemophilia (2001)
  • FIRST and ONLY site in Australia treating Thalassemia patients with gene therapy – initiated 2014
  • FIRST Australian site to treat patients with gene therapy for haemophilia A & B – until 2019 RPAH was the only site in Australia
  • FIRST site in NSW to be approved for commercial processing & distribution of Kymriah (CAR-T cell therapy) - June 2020
  • FIRST completed human trial of reprogrammed (iPSC-derived) cells (2020)

Manufacturing and Clinical Trial Services

Human Ingenuity Overcoming Human Frailty

Revolutionary technologies using human cells and genetic engineering for the development of novel therapies could provide cures for many human diseases - heart disease, organ failure and some cancers, as well as genetic diseases such as haemophilia and thalassaemia.

CMT provides services to academic and industry partners for development, manufacture, clinical evaluation and implementation of cell and gene therapies. We take discovery research into clinical practice with robust cell manufacturing protocols to meet Therapeutic Goods Administration (TGA) requirements for biologicals. Please contact the department for further details and pricing.

Our Objectives

  • Provide compliant manufacturing and clinical trial services to academic and industry partners
  • Support development, manufacture, clinical evaluation and implementation of novel cell and gene therapies
  • Provide improved treatment options for patients
  • Educate and train clinical, scientific, and regulatory staff

Our Capabilities

Clinical Trials

Our Clinical Trials team offers patients the opportunity of access to novel cell and genetic therapies, often when little to no alternative therapeutic options may be available. Our aim is to allow people to live longer, healthier lives and reduce hospital visits by helping develop new treatments. Utilising the services of our integrated cell processing laboratory allows us the opportunity to collect, manage, store, prepare and dispense investigational cell and gene therapy products. In addition to running our own Clinical Trials we also support Pharmacy and other trials at RPA with specialised storage of fully traceable patient specific products.


Operations are conducted in accordance with international, national, local and facility guidelines and regulations to ensure world class cell processing and preparation of quality cell and gene products. Our apheresis and transplant service were the first in NSW to be licenced by the TGA. We are also familiar with both U.S. Food & Drug Administration and European Medicines Agency requirements including PIC/s.

Process Development

Cell manufacturing processes are taken from research protocol through to clinically relevant scale in a separate process development laboratory. Staff training, documented processes and material and equipment qualification ensure that the developed processes will enable the manufacture of safe and consistent products in the cGMP environment. Process Development Laboratory infrastructure is supported with funds from the National Collaborative Research Infrastructure Strategy and Therapeutic Innovation Australia.


The self-contained, state of the art facility of over 400 m2 has been designed with flexibility to meet clinical research requirements. The manufacturing space includes four separate clean room suites, a large quality control and support laboratory, raw materials, and cell product storage facilities plus scientific and administrative support areas. The facility can accommodate the diverse needs of clinical academic researchers, as well as international biotechnology industry partners.

We have two large positive pressure, and two smaller negative pressure clean rooms to accommodate conventional and gene modification protocols. Isolated entry and exit gantries for each laboratory ensure unidirectional personnel flow. A central support laboratory provides common storage space and Quality Control testing facilities. All critical equipment used for product processing or storage is connected to a central environmental monitoring system that operates 24/7. Local visual alarms and remote alarms alert staff to ensure product quality and safety.

The facility was designed to be compliant with the Australian TGA standards for the manufacture of human cells for therapeutic use and the Office of the Gene Technology Regulator for clinical manufacturing protocols involving genetic modification. Revolutionary technologies using human cells and genetic engineering for the development of novel therapies could provide cures for many human diseases - heart disease, organ failure and some cancers, as well as genetic diseases such as haemophilia and thalassaemia.

Our Research Team

Dr Sharon Sagnella Research and Development Manager
Dr Michelle Keir Production Manager
Dr Aimei Lee Clinical Trials Manager
Jane Byrne Project Manager
Norvie Aquino Hospital Scientist
Laura Castelletti Hospital Scientist
Claire Chou Hospital Scientist
Luke Kleu Hospital Scientist
Sarah Nicholson Hospital Scientist
Fiona (Bao) Dang Technical Officer
Afroditi Sdrolias Logistics Coordinator
Jacinta Cable Clinical Research Coordinator
Miles Kenny Clinical Research Coordinator
Divya Suthar Clinical Research Coordinator
Rachel (RuiQiong) Zhao Clinical Research Coordinator
Craig Wright Quality Manager
Sharan Gopal Quality Officer
Mark Shannon Operations Manager
Luigia Manzoni Deputy Operations Manager
Mario Rodrigues Junior Administration Assistant

Rasko Lab

Understanding Disease – Finding a Cure

We are driven by the belief that the causes of disease must be understood at their molecular origins if rational treatments are to be designed. To understand cancer causation and the roots of genetic disease, we need to first understand how normal cells function.

Our primary goal is to integrate innovative cell and gene therapy research to improve human health. The Rasko Lab is comprised by the Gene & Stem Cell Therapy Program, the Li Ka Shing Cell & Gene Therapy Program, the Computational BioMedicine Lab and the Cancer & Gene Regulation Lab.

Selected Grants

Awardees Grant and project details
Rasko JEJ, Sandroussi C, Grimison P, Adelstein S. Cancer Council NSW, 2018-2022
Cellular immune-gene therapy for pancreatic cancer
Rasko JEJ Li Ka Shing Foundation, 2018-2022
Application of gene and cell therapy to the treatment of pancreatic cancer and potentially other mesothelin expressing cancers
Rasko JEJ Therapeutic Innovation Australia Limited (TIA), 2019-2023
In relation to the translating health discovery project
Rasko JEJ NHMRC, 2020-2024
Driving clinical cell and gene therapy in Australia
Rasko JEJ, Saxena P, Kench J, Yeo D, Sagnella S PanKind, 2022
Early detection and diagnosis of pancreatic cancer using circulating tumour cells

Our Publications

Latest Publications

Illuminating the dark protein-protein interactome. Tabar MS, Parsania C, Chen H, Su XD, Bailey CG, Rasko JEJ. (2022) Cell Reports Methods.
Approved gene therapies in Australia: coming to a store near you. Mallik S, Bailey CG, Rasko JEJ. (2022) Intern Med J.
Locoregional delivery of CAR-T cells in the clinic. Sagnella SM, White AL, Yeo D, Saxena P, van Zandwijk N, Rasko JEJ. (2022) Pharmacol Res.
20 Years of Legislation - How Australia Has Responded to the Challenge of Regulating Genetically Modified Organisms in the Clinic. O'Sullivan GM, Philips JG, Mitchell HJ, Dornbusch M, Rasko JEJ (2022) Front. Med.
Mapping oncogenic protein interactions for precision medicine. Tabar MS, Francis H, Yeo D, Bailey CG, Rasko JEJ (2022) Int. J. Cancer.
The next wave of cellular immunotherapies in pancreatic cancer. Yeo D, Giardina C, Saxena P, Rasko JEJ (2022) Mol. Ther. Oncolytics.
Exploring the Clinical Utility of Pancreatic Cancer Circulating Tumor Cells. Yeo D, Bastian A, Strauss H, Saxena P, Grimison P, Rasko JEJ. (2022) Int. J. Mol. Sci.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA (2021) N Engl J Med.
The Fusion of CLEC12A and MIR223HG Arises from a trans-Splicing Event in Normal and Transformed Human Cells. Dhungel BP, Monteuuis G, Giardina C, Tabar MS, Feng Y, Metierre C, Ho S, Nagarajah R, Fontaine ARM, Shah JS, Gokal D, Bailey CG, Schmitz U, Rasko JEJ. (2021) Int. J. Mol. Sci.
Mesenchymal Stromal Cells for the Treatment of Graft Versus Host Disease. Kelly K, Rasko JEJ. (2021) Front. Immunol.
Structure–function relationships explain CTCF zinc finger mutation phenotypes in cancer. Bailey CG, Gupta S, Metierre C, Amarasekera PMS, O'Young P, Kyaw W, Laletin T, Francis H, Semaan C, Sharifi Tabar M, Singh KP, Mullighan CG, Wolkenhauer O, Schmitz U, Rasko JEJ. (2021) Cell Mol Life Sci.
Splice and Dice: Intronic microRNAs, Splicing and Cancer. Wong ACH, Rasko, JEJ. (2021) Biomedicines.
Hitting the Bull’s-Eye: Mesothelin’s Role as a Biomarker and Therapeutic Target for Malignant Pleural Mesothelioma. Yeo D, Castelletti L, van Zandwijk N and Rasko JEJ. (2021) Cancers.
Unique protein interaction networks define the chromatin remodelling module of the NuRD complex. Tabar MS, Giardina C, Feng Y, Francis H, Sani HM, Low JKK, Mackay JP, Bailey CG, Rasko JEJ. (2021) FEBS J.
CTCF as a regulator of alternative splicing: new tricks for an old player. Alharbi AB, Schmitz U, Bailey CG, Rasko JEJ. (2021) Nucleic Acids Research.
Anti-Mesothelin CAR T cell therapy for malignant mesothelioma. Castelletti L, Yeo D, van Zandwijk N and Rasko JEJ. (2021) Biomarker Research.
Journey to the Center of the Cell: Tracing the Path of AAV Transduction. Dhungel BP, Bailey CG, Rasko JEJ. (2021) Trends in Molecular Medicine.

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