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Contact Details

  Street address:
Level 11, KGV Building
Missenden Road
Postal address:
Post Office Box M30
Missenden Road NSW 2050

Phone: (02) 9515-6111
Fax: (02) 9515-9610

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Cell and Molecular Therapies
Cell and Molecular Therapies

Cell and Molecular Therapies

Our Research

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 aim is to provide cellular therapies, regenerative medicines and tissue engineering based on a firm foundation of scientific evidence. We build on genetic technologies and disease models to understand the biology of adult stem cells and the causes of diseases including blood cancers, and genetic disorders.

Providing services to academic and industry partners for development, manufacture, clinical evaluation and implementation of cell and gene therapies.

Taking discovery research into clinical practice with robust cell manufacturing protocols to meet Therapeutic Goods Administration (TGA) requirements for biologicals.


Clinical grade processing

  • Haematopoietic progenitor cell (HPC) apheresis
  • Bone marrow harvest (BM)
  • Leukapheresis of mononuclear cells (MNC)
  • Isolation and expansion of Mesenchymal Stromal Cells (MSC)
  • Antigen presenting cells including dendritic cells (DC)
  • Tissue specific cells for regenerative medicine applications.
  • Cell processing
  • Cell expansion in static culture and bioreactors
  • Cryopreservation
  • Gene modification of cells using retroviral, lentiviral and other viral vectors.
  • Cell product characterisation by flow cytometry and PCR
  • Pharmaceutical formulation of viral vectors
  • Autologous serum eye drops

Conduct of clinical trials

  • Clinical trials - Phase I, II, III
  • Regulatory submissions to local and national bodies
  • Industry and Investigator initiated studies
  • Work to your IND or EU approved protocol
  • Fully compliant with ICH GCP standards
  • Experienced staff manage Study start-up to Close out
  • Long-term follow-up available

Clinical expertise

  • Cell - delivered gene therapies (thalassemia, haematological malignancies)
  • Viral vector gene therapies (haemophilia, hepatocellular carcinoma)
  • Immunotherapies (cancer, melanoma)


  • 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

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 Royal Prince Alfred Hospital with specialised storage of fully traceable patient specific products.


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.

The team led by Professor John Rasko has extensive cell and gene therapy expertise across all stages of product development. CMT focuses on product quality, through a combination of technical skills, project management, commitment to quality systems and operational excellence. CMT are committed to supporting the high quality research of collaborators in NSW, nationally and overseas. Manufacturing and research supported by CMT is performance driven to measurable outcomes.

Quality management

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.


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.

Key Achievements

  • CRC
  • TIA
  • Athersys
  • Terumo

Research Staff

Cell & Molecular Therapies RPAH:

  • Professor John Rasko, Director, Head of Department
  • Dr Stephen Larsen, Staff Specialist, Institute of Haematology, RPAH
  • Dr Janet Macpherson, Development Manager
  • Dr Zlatibor Velickovic, Production Manager
  • Dr Michelle Keir, Project Manager
  • Ms Natasha Barry, Hospital Scientist
  • Ms Flora Kan, Hospital Scientist
  • Ms Afroditi Sdrolias, Hospital Scientist
  • Mr James Favaloro, Hospital Scientist
  • Ms Linda Pallot, Registered Nurse


  • Amy Au - Research Officer
  • Amy Marshall - Research Officer
  • Carl Power - Editorial Research Officer
  • Chau-To Kwok - Research Officer
  • Chuck Bailey - Senior Research Officer
  • Cynthia Metierre - Research Assistant
  • Darya Vanichkina - Research Officer
  • Justin Wong - Associate Faculty
  • Maninda Amarasekera - Honours Student
  • Melissa Vellozzi - Research Assistant
  • Natalia Pinello - Research Assistant
  • Patrick O'Young - Research Assistant
  • Rajini Nagarajah - Research Assistant
  • Ulf Schmitz - Research Officer
  • Yue (Julie) Feng - Research Assistant

Higher Degree Students:


  • Frank Jia

Publications and Presentations

Publication Details:
  1. Weiss DJ, Rasko JEJ, Cuende N, Ruiz MA, Ho H-N, Nordon R, Wilton S, Dominici M, Srivastava A. Part 2: Making the 'unproven' 'proven'. Cytotherapy, Jan 2016, 18(1):120-123 IF 3.1 Cit: 1
  2. Wong JJ-L, Au AYM, Gao D, Pinello N, Kwok C-T, Thoeng A, Lau KA, Gordon JEA, Schmitz U, Feng Y, Nguyen TV, Middleton R, Bailey CG, Holst J, Rasko JEJ, Ritchie W. RBM3 regulates temperature sensitive miR-142-5p and miR-143 (thermomiRs), which target immune genes and control fever. Nucleic Acids Research, April 2016, 44(6):2888-2897 Cit: 1
  3. Edwards CR, Ritchie W, Wong JJ-L, Schmitz U, Middleton R, An X, Narla M, Rasko JEJ, and Blobel GAI. A dynamic intron retention program in the mammalian megakaryocyte and erythrocyte lineages. Blood, April 2016, 127(17) Cit:
  4. Keightley MC, Markmiller S, Love GC, Rasko JEJ, Lieschke GJ. Experimental approaches to studying the nature and impact of splicing variation in zebrafish. Methods in Biology ,[in press], doi:10.1016/bs.mcb.2016.02.006 Cit:
  5. Marshall AD, van Geldermalsen M, Otte NJ, Anderson L, Lum T, Vellozzi MA, Zhang BK, Thoeng A, Wang Q, Rasko JEJ, Holst J. LAT1 is a putative therapeutic target in endometrioid endometrial carcinoma. International Journal of Cancer, 2016 [in press]

Book Chapters:
  1. Keightley MC, Markmiller S, Love CG, Rasko JEJ, Heath JK, Lieschke GJ. Experimental approaches to studying the nature and impact of splicing variation in zebrafish in The Zebrafish: Genetics, Genomics and Transcriptomic, Fourth Edition, Methods in Cell

  1. 'Cell therapy in Asia' 6th Annual Bio Manufacturing Conference 2016, Grand Hyatt Shanghai, China, 16-19 May 2016
  2. 'From cells to genes in one delicious mouthful', UTAR, Faculty of Medicine and Health Sciences, Sungai Long Campus, Selangar, Malaysia, 23 May 2016
  3. '...', University of Cordoba, Faculty of Science, Spain, 11 & 12 July 2016
  4. 'Intronic nonsense: From gene expression to cancer' The John Curtin School of Medical Research School Seminar Series, Australian National University College of Medicine, Biology & Environment, Canberra, 29 July 2016
  5. '....'Olivia Newton-John Cancer Research Institute, Heidelberg, Melbourne, 11 August 2016
  6. ' .....' Bellberry Education Weekend 2016, Adelaide, 14 August 2016
  7. 'Progress in gene therapy for genetic diseases', Ritchie Centre Colloquium, Hudson Institute of Medical Research, Clayton, Melbourne, 25 August 2016
  8. 'Cell Therapy Update: Disentangling hope, realism and quackery', Ritchie Centre Colloquium, Hudson Institute of Medical Research, Clayton, Melbourne, 26 August 2016

Research Grants

Clive and Vera Ramaciotti Foundations Centenary Institute The Ramaciotti Centre for Human Systems Biology  $1,000,000 2014-2016
National Health & Medical Research Council Rasko Intron retention regulation in granulopoiesis and leukaemia  $565,280 2014-2016
National Health & Medical Research Council Ritchie/Rasko Investigating widespread regulation of gene expression through intron retention  $354,549 2015-2017
National Health & Medical Research Council Winkler/Rasko Haematopoietic Stem Cell glycome regulates outcome of niche interactions  $131,832 2015-2018
New South Wales Cancer Council Rasko Consequences of CTCF haploinsufficiency in endometrial carcinoma  $360,000 2014-2016
New South Wales Cancer Council Rasko Consequences of CTCF mutation in acute lymphoblastic leukaemia  $360,000 2014-2016
New South Wales Cancer Council Holst Starving Cancer Cells: Developing nutrient uptake inhibitors as prostate cancer therapeutics  $360,000 2015-2017
Anonymous Rasko  n/a  $3,164,640 2013-2017
Anonymous Rasko/Macpherson Cell & Molecular Therapies  $402,000 2013-2017
Anonymous Rasko/Macpherson CRC for Cell Therapy Manufacturing  $250,000 2015-2019

Contact details for department

Head of Department: Professor John E J Rasko

Department/Unit: Cell and Molecular Therapies
RPA Hospital
Missenden Road
Camperdown NSW 2050
Telephone: (02) 9515 4860
Facsimile: (02) 9515 4868
Web: www.slhd.nsw.gov.au/research/
Email: janet.macpherson@health.nsw.gov.au