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. Please contact the department for further details and pricing. Taking discovery research into clinical practice with robust cell manufacturing protocols to meet Therapeutic Goods Administration (TGA) requirements for biologicals.

Capabilities

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)

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

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.

Operations

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. Process Development Laboratory infrastructure is supported with funds from the National Collaborative Research Infrastructure Strategy and Therapeutic Innovation Australia.

Facilities

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 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. Please contact the department for further details and pricing. Taking discovery research into clinical practice with robust cell manufacturing protocols to meet Therapeutic Goods Administration (TGA) requirements for biological.

Awards/Medals

Professor John Rasko. Research Excellence Award, Sydney Innovation and Research Symposium, SLHD
Professor John Rasko. Donald Metcalf Oration, New Directions in Leukaemia Research Conference, Brisbane - 2018.


Research Staff

Cell & Molecular Therapies RPAH:

  • Professor John Rasko, Director, Head of Department
  • 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
  • Mr Samuel Gardiner, Registered Nurse
  • Ms Merlin Thomas, Registered Nurse
  • Ms Divya Suthar, Registered Nurse

CENTENARY:

  • Adel Alharbi - PhD Student
  • Alexandra Aicher - Senior Research Officer
  • Bijay Dhungel - Research Officer
  • Carl Power - Editorial Research Officer
  • Caroline Giardina - Honours Student
  • Chuck Bailey - Senior Research Officer
  • Crystal Semaan - Research Officer
  • Cynthia Metierre - Research Assistant
  • Dannel Yeo - Research Officer
  • Geoffray Monteuuis - Research Officer
  • Gerard Chu - PhD Student
  • Habib Francis- PhD Student
  • Jaynish Shah - Research Officer
  • Mia Nguyen - Honours Student
  • Mehdi Taber - Research Officer
  • Melissa Vellozzi - Research Assistant
  • Paddy Kearney - Research Assistant
  • Rajini Nagarajah - Research Assistant
  • Ulf Schmitz - Research Officer
  • Veronika Petrova - Honours Student
  • William Wang - BSc(Med) Honours Student
  • Yue (Julie) Feng - Research Assistant

Publications and Presentations

Publication Details:

1.     George, L. A., Sullivan, S. K., Giermasz, A., Samelson-Jones, B. J., Ducore, J. M., Teitel, J. M., et.al. (2018). SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B-Sustained Fix Activity, Persistent Endogenous Prophylaxis and Improved Quality of Life at One Year and Beyond. In Molecular Therapy: 26, 1.

2.     Thompson, A. A., Walters, M. C., Kwiatkowski, J., Rasko, J. E. J., Ribeil, J.-A., Hongeng, S., Cavazzana, M. (2018). Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia. New England Journal of Medicine, 378(16), 1479-1493.

3.     George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., ... High, K. A. (2017). Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. New England Journal of Medicine, 377(23), 2215-2227.

4.     Vanichkina DP, Schmitz U, Wong JJL, Rasko JEJ. Challenges in defining the role of intron retention in normal biology and disease. Seminars in Cell & Developmental Biology, 2018 75:40-49. Wong A, Rasko JEJ, Wong JJ. We skip to work: alternative splicing in normal and malignant myelopoiesis. Leukemia 2018 32(5):1083-1093.

5.     Sluys R, Vila-Farre M, Rink J, Rasko JEJ. An intriguing, new planarian species from Tasmania, with a discussion on protandry in triclad flatworms (Platyhelminthes, Tricladida). Acta Zoologica 2018; 1-11 Zhao G, Bailey CG, Feng Y, Rasko JEJ, Lovicu FJ. Negative regulation of lens fiber differentiation by RTK antagonists Spry and Spred. Experimental Eye Research, 2018 170:148-59.

6.     Sipp D, Frazer IH, Rasko JEJ. No vacillation on HPV vaccination. Cell, Leading Edge, Commentary, 2018 8;172(6):1163-1167.

7.     Rajasekhar M, Schmitz U, Flamant S, Wong JJ, Bailey CG, Ritchie W, Holst J, Rasko JEJ. Identifying microRNA determinants of human myelopoiesis. Scientific Reports 2018 May 8;8(1):7264.

8.     Jaenisch R, Dubois N, Rasko JEJ, Hongkui D, Alvarado A, Fuchs E, Vunjak-Novakovic G, Baldwin, K. Challenging Stem Cells. Cell, Leading Edge. Commentary 2018 173(5):1063.

9.     Nair S, Luu P-L, Qu W, Maddugoda M, Huschtscha L, Reddel R, Chenevix-Trench G, Toso M, Kench JG, Horvath LG, Hayes VM, Stricker PD, Hughes TP, White DL, Rasko JEJ, Wong JJ-L, Clark SJ. Guidelines for whole genome bisulphite sequencing of intact and FFPET DNA on the Illumina HiSeq X Ten. Epigenetics & Chromatin 2018 May 28:11(1):24.

10.     Sandt C, Feraud O, Bonnet ML, Meunier MC, Khedir R, Bertrand A, Flamant S, Bailey CG Rasko JEJ, Dumas P, Bennaceur-Griscelli A, Turhan AG. Direct and rapid identification of T315I-mutated BCR-ABL expressing cells using infrared microspectroscopy. Biochemical and Bioresearch Communications 2018 Sep 10;503(3):1861-1867.

11.     Rasko JEJ & Priess T. Diversity of transcripts emanating from protein-coding genes. Seminars in Cell and Developmental Biology 2018 75:1-2.

12.     Cuende N, Rasko JEJ, Koh MBC, Dominici M, Ikonomou L. Cell, tissue and gene products with marketing authorization in 2018 worldwide. Cythotherapy 2018 Nov;20(11):1401-1413.

13.     Bailey CG, Metierre C, Feng Y, Baidya K, Filippova GN, Loukinov DI, Lobanenkov VV, Semaan C, Rasko JEJ. CTCF expression is essential for somatic cell viability and protection against cancer. Int J Mol Sci. 2018 Nov 30;19(12).


Presentations:

1.     Bloor, A., Patel, A., Griffin, J. E., Gilleece, M. H., Radia, R., Yeung, D. T., ... Rasko, J. E. J. (2018). A Phase I Trial of iPSC-Derived MSCs (CYP-001) in Steroid-Resistant Acute GvHD. Blood, 132(Suppl 1), 4562-4562. https://doi.org/10.1182/BLOOD-2018-99-110432.

2.     Porter, J., Walter, M., Kwiatkowski, J., Rasko, J. E. J., Hongeng, S., Schiller, G. J., Thompson, A. (2018). Safety and efficacy of Lentiglobin Gene Therapy for Transusion-Dependent β-thalassemua (TDT) in Patients of non-β0/β0 Genotypes: Interim results of the HGB-204 (Northstar) and HGB-207 (Northstar-2) Trials. In British Society of Haematology Meeting. Liverpool.

3.     Walters, M. C., Kwiatkowski, J. L., Rasko, J. E. J., Hongeng, S., Schiller, G. J., Anurathapan, U.,  Thompson, A. A. (2018). Safety Following Autologous Transplantation with Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in the Northstar Hgb-204 Study. Biology of Blood and Marrow Transplantation, 24(3), S74. https://doi.org/10.1016/j.bbmt.2017.12.643.

4.     Rasko, J. E. J., Thompson, A. A., Kwiatkowski, J. L., Hongeng, S., Schiller, G. J., Anurathapan, U., ... Walters, M. C. (2018). Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study. Blood, 132(Suppl 1), 167-167. https://doi.org/10.1182/BLOOD-2018-167.

5.     Porter, J., Walter, M., Kwiatkowski, J., Rasko, J. E. J., Hongeng, S., Schiller, G. J., Thompson, A. (2018). Safety and efficacy of Lentiglobin Gene Therapy for Transusion-Dependent β-thalassemua (TDT) in Patients of non-β0/β0 Genotypes: Interim results of the HGB-204 (Northstar) and HGB-207 (Northstar-2) Trials. In British Society of Haematology Meeting. Liverpool.


Research Grants

Granting Body

Awardees

Project Title

Grant total $

Years

Cancer Council NSW

Rasko JEJ

Cellular immune-gene therapy for pancreatic cancer

 $2,000,000

2018-2022

Li Ka Shing foundation

Rasko JEJ

Application of gene and cell therapy to the treatment of pancreatic cancer and potentially other mesothelin expressing cancers.

 $4,497,500

2018-2022

Contact details for department

Head of Department: Professor John Rasko

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

 

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