CRC for Cell Therapy ManufacturingAnnual Report

2016

Table of Contents6Chair’s Report

5CEO’s

Certification

8Executive Summary

16Education &

Training

12Research SME

Engagement

20

26Marketing &

Communications

22Utilisation &

Commercialisation Governance

30

40 Participants

36Key Staff Collaboration

42

46 Publications & Presentations

44Financial

ManagementGlossary of

Terms

48

CRC BoardDr Leanna Read (Chair)Dr Stephen LiveseyDr Alexander Gosling AMMr Charlie LathamMr Ray WoodDr Sherry Kothari ParticipantsAthersys, Inc.

Cell Therapies Pty Ltd

Exylika Pty Ltd

Medvet Science Pty Ltd

NextCell Pty Ltd

Queensland University of Technology

The Royal Adelaide Hospital - a division of Central Adelaide Local Health Network, Inc.

Royal Prince Alfred Hospital (Sydney Local Health District)

St Vincent’s Institute of Medical Research

SA Pathology - a division of Central Adelaide Local Health Network, Inc.

South Australian Health and Medical Research Institute (SAHMRI) Ltd

Terumo BCT, Inc.

University of South Australia

University of Sydney

University of Wollongong

Women’s and Children’s Health Network Inc.

The CRCThe CRC for Cell Therapy Manufacturing (CTM CRC) is facilitating the cost-effective manufacture and rapid translation of cell therapies into clinical practice.

CTM CRC has a total of $60M in cash and in-kind resources, including a $20M grant from the Australian Government over six years. CTM CRC is providing new treatments and developing new materials-based manufacturing technologies for the treatment of conditions such as immune-mediated diseases, chronic wounds, diabetes and cardiovascular diseases.

Headquarted in Adelaide, South Australia, CTM CRC brings together the spectrum of skills and facilities required to turn promising cell-based technologies into viable cell therapies. CTM CRC’s national and international participants include research providers, manufacturers, cell therapy companies, hospitals and charities.

Underpinning this partnership is a cGMP compliant facility, designed to test and validate new processes, technologies and therapies for commercial scale manufacturing.

VisionTo provide new treatments and develop new materials-based manufacturing technologies to increase the accessibility, affordability and efficacy of cell therapies.

Head OfficeLevel 5Catherine Helen Spence BldgUniSA City West CampusAdelaide, South AustraliaAUSTRALIA 5000

T +61 8 8302 3503E info@ctmcrc.comW ctmcrc.com

ABN 92 163 117 520

twitter.com/CTMCRC facebook.com/ctm.crc

linkedin.com/company/crc-for-cell-therapy-manufacturing

CRC for Cell Therapy Manufacturing Annual Report 2016 32

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I hereby certify that the information provided to the Department of Industry, Innovation and Science by the CRC for Cell Therapy Manufacturing in:

n the written annual report for the 2015-16 financial year;

n the CRC’s online milestone tables for the 2015-16 financial year;

n the CRC’s online financial tables for the 2015-16 financial year;

n the CRC’s online MDQ for the 2015-16 financial year; and

n the four quarterly reports provided online for the 2015-16 financial year

is accurate and provides a true and fair view of the matters reported on therein.

I certify that the Commonwealth Funding and Participant Contributions were applied for the Activities of the CRC as specified in the Commonwealth Funding Agreement and that Commonwealth Funding has been expended only for the Activities and otherwise in accordance with this Agreement.

I certify that the CRC has met its obligations in relation to the treatment of intellectual property.

I certify that the chair of the board meets the requirements of independence set out in the Commonwealth Agreement and that the majority of board members are independent of the CRC’s research providers.

I certify that the CRC has adhered to the requirements for proper use of the CRC Programme indicia, publicity and support as specified in the Commonwealth Funding Agreement.

I am aware that giving false or misleading information is a serious offence and could lead to prosecution under the Criminal Code 1995.

Dr Sherry KothariCEO & Managing Director, CRC for Cell Therapy Manufacturing27/10/2016

CEO’s Certification

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Last year, I predicted that 2015-16 would be an important year for CTM CRC. By the end of our third year, we intended to identify and focus our resources on those projects with the highest probability of achieving commercial application within the short to medium term. This strategy underpins CTM CRC’s transition planning to achieve sustainability after funding from the Federal CRC Programme ceases.

Transition planning has since been put into sharp focus with release of the Miles Review into the CRC Programme, and the subsequent announcement by the Federal Government that no existing CRC would receive follow-on funding. This decision came as somewhat of a surprise to the CRC sector, but I am pleased that our proactive approach has put CTM CRC on the front foot.

CTM CRC has been an industry-focused CRC from day one, with one overriding focus – to solve the rate-limiting problems of the global cell therapy industry related to the high costs of cell manufacture.CTM CRC’s remaining three years will have three key focus areas, designed to position CTM CRC towards sustainability at the end of our six-year funding period. These include materials and interfaces,

pilot scale manufacturing and training, and cellular immunotherapies.

Novel materials and interfaces underpin CTM CRC’s approach to reduce the cost of cell therapy manufacture, formulation and delivery. The current high cost of these processes is a major impediment to bringing cell therapies into mainstream medicine. We have already made excellent progress with new low-cost surface coatings, lattices and capture technologies for expansion of several cell types. Other developments include biomaterials and surface coatings to deliver cells to injury sites, such as wounds and for anti-fouling of stent implants. CTM CRC is already in discussions with a number of global commercial entities regarding some of these opportunities. Hence, the remaining three years will allow us to refine the technologies and position us to market a range of patent-protected, globally competitive processes to the international cell therapy industry.

One of the marketing strategies for these novel materials and interfaces will be to offer pharmaceutical and biotechnology industry clients services to optimise their cell therapy manufacturing for commercial scale. For this purpose, we are establishing an Australian prototyping facility that will provide a range of platforms and biomaterials for developing and integrating our novel interfaces into clients’ final, current Good Manufacturing Practice (cGMP)-compliant manufacturing protocols. The intent is to become the premier ‘go-to’ prototyping facility of its kind in Australia and worldwide. Besides operating as a commercial prototyping facility, we will offer training courses to address the world-wide shortage of skills in operating cGMP clean room manufacture in the cell therapy industry.

As our final strategy towards sustainability, we have established a spin-out company, Carina Biotech Pty Ltd, to exploit a suite of patented CRC technologies in the global T cell immunotherapy industry. Immunotherapy is one of the largest and fastest growing sectors of the cell therapy market. In particular, a new approach that uses Chimeric Antigen Receptor T cells (CAR-T cells) is showing unprecedented promise for treatment of some types of leukemia. CTM CRC is developing technologies spanning a new CAR-T cell that may target and kill other cancers other than leukaemia, specifically solid tumours; improved methods for manufacturing CAR-T cells; lattices for effective T cell expansion, and materials for delivering and retaining cells at tumour sites. We anticipate that Carina Biotech will be well placed within the next years to secure substantial investment capital to position it for rapid growth as a promising new Australian biotechnology company.

Returning to the Miles Review, the CTM CRC board was encouraged by the recognition that “the CRC Programme continues to be extremely important in encouraging and facilitating industry-led collaboration between industry and research”. I am confident that the directions and focus of CTM CRC are entirely consistent with the new CRC Programme priorities, particularly the key recommendation to put industry ‘front and centre’.

CTM CRC now has a range of exciting opportunities, supported by rigorous processes to position us towards sustainability after our final three years of CRC Programme funding. This will, in turn, help to achieve the Miles Review imperative that CRCs should lead to “increased jobs, exports, productivity, integration into global supply chains, new technologies, products or services, increased revenues and intellectual property outputs such as patents.”

In closing, I would like to thank the CTM CRC Board, and particularly Managing Director, Dr Sherry Kothari, for their passion and leadership over the last year. I also congratulate the CTM CRC management and the research project leaders for their pivotal role in building the vision for the next three years.

Dr Leanna ReadChair, CTM CRC

Dr Leanna ReadChair, CTM CRC

Chair’s Report

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The rapid growth in the field of regenerative medicine, and cell therapies in particular, has provided CTM CRC with an ideal and timely opportunity to put Australia at the vanguard of cell therapy manufacture. Three years ago, Australia had a small, fragmented cell therapy community, with a limited international footprint. This was despite outstanding research capability, expertise and infrastructure.

Through CTM CRC, we are consolidating this expertise and putting Australia very firmly on the global map. In 2017, in collaboration with one of our US-based industry participants, our first technology output will take a therapy for the treatment of chronic diabetic foot ulcers into the clinical phase. Highlights and achievements in the last year include:

n Three new provisional patent applications covering T cell expansion, a CAR-T cell and a surface-modified anti-fouling stent

n One international patent application for a wound repair delivery system

n CTM CRC’s first spin-out company, Carina Biotech Pty Ltd, with a focus on immunotherapies

n Initial commercial beta testing of CTM CRC’s T cell expansion technology at the Cell and Gene Therapy (CGT) Catapult, UK

n Evaluating T cell expansion lattice in Wilson Wolf’s G-Rex system

n Attracted over $1M of new funding

CTM CRC recently undertook a strategic planning exercise, which was an iterative process involving consultation with participants, researchers and key industry opinion leaders.

Shaped by a maturing global landscape, unprecedented clinical results with immunotherapies, and an increasing focus on scale-up and manufacturing, CTM CRC’s key focus areas build on progress to-date, core capabilities and industry pull. CTM CRC’s renewed focus allows for synergy across platform technologies, therapeutic applications and commercial objectives. CTM CRC is ‘cell agnostic’ in its approach to reducing the cost of goods, providing a strategic advantage and flexibility to adapt, should a different cell become more commercially relevant. CTM CRC’s strategy going forward will thus have three focus areas:

Pilot scale-up and training facilityThe demonstration of scalability, regulatory compliance and manufacturability is a logical next step for technology development, before progressing to commercial exploitation. As we develop new technologies to solve manufacturing challenges, this facility will help to navigate the translation of technologies from laboratory to pre-commercial scale. CTM CRC sees this as an important and vital transition in its evolution to achieving success.

In collaboration with organisations such as the Cell and Gene Therapy Catapult, UK and GE Healthcare, CTM CRC is also working on developing specific training modules that can be harmonised globally. These will be dovetailed with the pilot scale-up facility to establish a training hub that will place Australia at the heart of an emergent global industry.

Cellular immunotherapiesThere is currently a dominant focus on immunotherapy developments within the industry. CTM CRC’s T cell expansion technology has been shown to be competitive with the industry gold standard at the laboratory scale, and is currently being tested in a commercial system in collaboration with a US company. The technology is also being evaluated for scale-up through an agreement with CGT Catapult. With a range of other complementary technologies in development, including tumour-specific targeting and cell delivery mechanisms. CTM CRC has formed a spin-out company, Carina Biotech Pty Ltd, which will seek to exploit opportunities through the development and commercialisation of T cell related IP assets.

Materials and interfacesCTM CRC’s expertise in materials and surface manipulation is being increasingly recognised as an internationally competitive and much needed resource for the cost-effective manufacture and delivery of cell therapies. This area captures demonstrated and available capability that contributes to improvement in expansion, formulation and delivery of therapeutic cells.

AchievementsAwardsForget, A - Student & Young Investigator Section Poster Award 1st Place2016 Tissue Engineering & Regenerative Medicine International Society (TERMIS), Asia Pacific meeting

Griesser, H - Award for Research ExcellenceAustralasian Society for Biomaterials & Tissue Engineering

Smith, L - Early Stage Professional Award2015 International Society for Cellular Therapy (ISCT) ANZ Regional Meeting

Weiss, A - Fellow of Biomaterials Science and Engineering (FBSE) The International Union of Societies for Biomaterials Science & Tissue Engineering

Dr Sherry KothariCEO & Managing Director, CTM CRC

Executive Summary

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Weiss, A - Member-At-Large – Election to CouncilTERMIS

Weiss, A - Fellow of the Royal Australian Chemical Institute (FRACI)Royal Australian Chemical Institute

Weiss, A - Innovator of Influence Award2015 Australian Science and Innovation Forum

Weiss, A - Applied Research MedalRoyal Australian Chemical Institute

Research & Collaboration n University of Wollongong joined CTM CRC to

specifically explore bioprinting utility in cell therapy

n Positive pre-clinical data for wound healing with cell delivery device progressing towards first-in-man clinical trials

n Prototype technology developed for more cost-effective manufacture of T cell therapies

n A novel coating for vascular stents demonstrates positive anti-fouling properties in preclinical testing

n Two collaborative projects commenced through international linkages formed in the previous year

n New tumour target and gel-based delivery of cells in development

Commercialisation & Utilisation n CTM CRC continues to grow a strong patent

pipeline and its IP model provides an easy, obstacle-free pathway to transact deals and exploit IP

n Three new provisional and one international patent applications lodged

n First CTM CRC spin-out company formed

Education & Training n The first joint PhD studentship between University

College London (UCL) and CTM CRC commenced

n Four new PhD students and two Honours students, bringing total CTM CRC PhD students to 15

n Two entrepreneurial PhD (ePhD) Programme in-residence workshops delivered

� Startup! Bootcamp

� PhDs@work (business skills)

Communications n Co-hosted the 2015 ISCT ANZ Regional Meeting -

‘Translating Cell Therapy 2015’

n CTM CRC’s inaugural two-day ImpaCT Workshop held

Risks & ImpedimentsCTM CRC’s recent strategic review was instigated, in part, by the outcomes from the last CRC Programme Review and coincided with CTM CRC’s mid-term. The main objectives of the strategic review were to benchmark all existing and future projects against unmet clinical or industry needs and prioritise funding to promote utilisation. This will help with future planning towards sustainability.

The strategic review was conducted with wide consultation, ensuring buy-in and continued participant and researcher engagement. Through this process, personnel crucial to the delivery of the new plan were identified, and the retention of these key personnel will be a CTM CRC priority.

CTM CRC’s Audit & Risk Committee continues to oversee matters relating to due care, diligence and conflict and plays a vital role in risk evaluation and control.

End-user EnvironmentThe cell therapy industry continues to show dramatic increases in financing and merger and aquisition activities. Total capital raised in the area of regenerative medicine more than doubled from $5.2bn to $10.8bn during the reporting period. The number of clinical trials in the cell therapy industry rose by 30%. T cell-based immunotherapies remained the most investigated in the industry, driven by the clinical successes of CAR-T cell therapies, with more than 40% of all cell-based clinical trials involving immunotherapies.

Many countries and regions now have well-established, private and public sector organisations that are focused on addressing the major challenges facing development of cell-based therapies. Challenges include the high cost of manufacture, evolving regulatory frameworks, reimbursement strategies and supply logistics.

CGT Catapult and the Centre for Commercialization of Regenerative Medicine (CCRM) are examples of such initiatives in the UK and Canada, respectively. CTM CRC has well established strategic alliances with both organisations and is establishing similar links with organisations in Asia.

These strategic alliances have led to a greater understanding of each organisation’s capabilities and a willingness to capitalise on synergies to create new collaborative opportunities that impact the industry. From these discussions, a collaborative project has already been initiated with the CGT Catapult. In addition, CTM CRC is co-developing an industry training programme with them.

The CTM CRC Board and management conduct regular strategic discussions to stay abreast of developments in the field. Additionally, CTM CRC regularly engages with key opinion leaders and subject matter consultants to ensure the currency of its research and outputs.

CTM CRC has increased its profile in the industry through frequent conference presentations and attendances at key meetings. This has led to a number of Australian and overseas organisations expressing an interest in CTM CRC capabilities and potential collaboration opportunities:

n Cell therapy companies with a proprietary cell or those targeting a specific clinical indication e.g. Athersys, Inc., Juno Therapeutics

n Cell therapy tools companies, including those providing reagents and hardware e.g. Terumo BCT, Inc., Merck-Millipore, St Gobain

n Companies that specialise in scale-up, automation and large-scale manufacture e.g. GE Healthcare, Invetech, Miltenyi Biotec

n SMEs that are positioned to exploit technological platforms e.g. Cell Therapies Pty Ltd

n Clinics, hospitals and patients

Impacts n In collaboration with one of CTM CRC’s industry

participants, a therapy for the treatment of chronic wounds is currently in pre-clinical phase. On the basis of promising results, design and planning for clinical studies in Australia is underway and could soon deliver a therapy for patients with diabetic foot ulcers

n CTM CRC has developed a novel material for the rapid expansion of T cells that will improve the cost and efficiency of this process. The material is designed to integrate into existing commercial platforms and CTM CRC is working closely with a US company to evaluate it in their system

n CTM CRC has also started a technology transfer evaluation of the material in collaboration with the

CGT Catapult, which has the capacity to compare the performance of the material across a range of platforms prior to commercial scale manufacture

n CTM CRC is exploring a novel target that could form the basis of a cancer therapy for a range of cancers, particularly solid tumours. Proof-of-concept data will be generated over the next twelve months

n CTM CRC has developed a novel technology that may reduce fouling and thrombosis in stents. This technology is being tested for proof-of-concept with a range of commercially available stent materials. CTM CRC is in discussions with a large multinational pharmaceutical company interested in the technology

n The establishment of a pilot scale-up facility is an important utility that will facilitate the seamless integration of promising technologies into commercial systems. An invaluable test-bed for technology development, the facility will provide proof-of-concept data and validation for emerging technologies

n The scale-up facility will also provide an ideal environment for the hands-on training of industry personnel who wish to familiarise themselves with the principles of cell therapy manufacturing, and with cell-specific bioreactor platforms. Pilot training modules will be developed in collaboration with organisations such as the CGT Catapult, with industry input provided by companies such as GE Healthcare

CTM CRC researcher, Dr Giles Kirby

Executive Summary

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Highlights n 21 of 25 milestones were achieved

n Three new provisional patent applications and one PCT application lodged

n cGMP facility established for scale-up manufacture of cell-based therapies and cGMP training

n Five new research projects were initiated

CTM CRC remains focused on its vision to develop innovative technologies and materials-based solutions to increase the accessibility, affordability and efficacy of cell-based therapies.

CTM CRC is in a strong position to become a key contributor to the local and global cell therapy industries and has engaged a number of commercial companies with interest in CTM CRC developed technologies.Performance against activitiesFive new projects commenced, increasing the total number to fourteen. Two projects were completed, and all remaining projects are progressing well.CTM CRC’s quarterly regulatory review of projects helps identify risks and guides project teams in the

development of strategies to minimise potential delays and incorporate additional technical requirements. Total product profiles for all projects have been generated as an outcome of these activities.

Of the 25 milestones from this reporting period, 21 were completed and it is expected that two of the four in-progress milestones will be completed in the next reporting period. One milestone is unlikely to be completed due to a change in project direction. Research publication milestones were delayed to allow for lodgement of patent applications. Up to 14 manuscripts are currently in progress and will be submitted for publication in the next reporting period.

Programme 1: Materials & Bioprocessing This programme involves the development of new materials and surfaces to facilitate the cost-effective manufacture and delivery of therapeutic cells. Focusing on bioprocessing systems, the programme is investigating platform technologies to improve the isolation, expansion and delivery of cells. The programme’s key research includes the design and development of:

n New surface coatings for bioreactors to improve the activation and expansion of cells

n Novel capture technologies to facilitate target cell isolation and expansion

n New biomaterials and advanced surface coatings to deliver cells to the site of injury, such as diabetic wounds

n Lattices and polymers for the co-culture and delivery of combinations of therapeutic cells

Activities in Programme 1 Improving manufacturing cell yieldsCell expansion is a major cost in the manufacture of a cell therapy and an impediment to successful clinical translation. CTM CRC researchers have successfully developed improved processes for expanding therapeutic cells. The processes are adaptable to multiple cell types and bioreactor systems and have the potential to reduce the cost of goods for cell therapies.

A key challenge with new technologies and processes is their successful integration into existing hardware

or tools currently used by companies. CTM CRC is focused on translating successful technologies developed at the laboratory-scale to demonstrate proof-of-concept at commercial scale. Surfaces for expansion of therapeutic cells Cells such as human regulatory T cells (Tregs) and endothelial progenitor cells (EPCs) have the potential to be used as adjunct therapies in organ and cell transplantation, however, significant challenges remain. Target cells need to be isolated from an individual patient for autologous use. Following isolation, cells must be expanded significantly over a short period of time, often starting from low numbers.

By combining novel manufacturing techniques, surface optimisation and high-throughput screening, CTM CRC researchers have developed prototype materials that induce the rapid expansion of Tregs and T effector

cells, such as those used in CAR-T cell therapies. The subject of a new provisional patent application, this technology has the potential to reduce the cost and complexity of manufacture of T cell-based therapies in a variety of bioreactor platforms.

There has been significant interest in this technology from a number of bioreactor device suppliers. The technology is now being evaluated in commercial expansion systems through collaborations with individual companies, as well as organisations such as CGT Catapult.

Endothelial progenitor cells have been used to screen for key bioactive molecules, with the potential to selectively expand them for therapies to treat acute and chronic cardiovascular conditions. Translation of this work from the laboratory to proof-of-concept has been initiated to determine its impact on cost and supply. This activity has also led to a novel antithrombotic coating for stents and is the subject of another provisional patent application. CTM CRC is currently in discussions with a major global supplier of coronary and vascular stents about utilisation and potential commercialisation of this technology.

Pluripotent stem cells (PSCs) can be used to make virtually any cell type and are gaining interest as ‘an endless supply’ of many therapeutic cell types. These cells require very expensive raw materials and CTM CRC has initiated a pilot project to apply the use of advanced surfaces to reduce the costs associated

CTM CRC and cellular immunotherapies

n Strong presence in the cellular immunotherapy arena

n Complementary technologies being developed

n Most advanced of these addresses cost effective T cell expansion

n Currently being tested in a range of proprietary expansion platforms to allow for integration into these systems

n Interest from at least two global suppliers of T cell expansion equipment that will provide a path to market

n Evaluation of technology transfer and commercial feasibility commenced in partnership with CGT Catapult

n Other technologies in development include formulations for the delivery of immunotherapies to tumours and materials-based platforms to improve efficiency of genetic modification of T cells for the treatment of a variety of conditions.

Research

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with PSC culture and differentiation. The success of this project is likely to lead to subsequent projects to extend the application of the technology to other cell types. CTM CRC is in discussions with a commercial company about these opportunites. Two further projects, with the University of Nottingham and UCL, have also been initiated to investigate novel coatings for cost effective expansion and conversion of PSCs to therapeutic cells.

A cell delivery device for chronic diabetic wounds Chronic wounds are a significant burden on healthcare costs. During the last reporting period, CTM CRC researchers successfully developed a dressing to deliver therapeutic cells to wounds, leading to a provisional patent application filing in early 2015. In collaboration with the project’s commercial participant, a first round of prototype testing to determine a preferred composition and treatment has been completed.

Preclinical testing has confirmed early positive results and a PCT has now been lodged. The preferred device material and coating combination is now well advanced in its evaluation for safety and efficacy.

Lattices for the transport and delivery of human islets to treat diabetes The number of cases of Type 1 diabetes in Australia is expected to double over the next twenty years. Donor islet transplantation is a promising treatment and a potential cure for diabetics. Existing methods

for isolation yield low numbers of viable islets. CTM CRC researchers are developing devices that aim to maximise islet survival during the critical 24-hour period following isolation.

Prototype transport devices with surface features have been shown to improve islet function. There is potential for such devices to improve the availability of donor islets for patients. CTM CRC is currently focusing its efforts on technical challenges associated with the manufacture of prototypes for testing.

A T cell-based immunotherapy A four-month pilot study was undertaken to investigate a novel CAR-T cell. The new CAR-T cell harnesses a patient’s own immune system to attack solid cancers.This work builds upon the effectiveness of previous clinical studies demonstrating CAR-T activity against leukaemia. CTM CRC has now secured additional funding for two years to further investigate this therapy. The project has already gained the interest of leading CAR-T development companies seeking novel cancer targets and is the subject of a provisional patent application.

Discussions with a key opinion leader in the CAR-T field have also led to a pilot project to investigate the utility of a thermo-responsive hydrogel for delivery of CAR-T cells to solid tumours. The work is likely to lead to a potential collaboration with a US-based clinical research institution.

Programme 2: Clinical Translation This programme aims to progress development and clinical translation of promising technologies from Programme 1 and includes:

n Preclinical safety and efficacy studies to generate data packages towards readiness for clinical trials

n Establishment of a GMP compliant facility for pilot scale-up as a test bed to validate technologies and cell therapies for human clinical testing

n Alignment with regulatory requirements and protocols for products or technologies developed by CTM CRC

n Development of prototype products and pilot scale manufacture of investigational clinical material to cGMP standards

n Development of human research ethics submissions and planning of human clinical studies

Activities in Programme 2 Generation of preclinical data Conducting preclinical trials using appropriate models of disease is essential in developing a cell therapy. These trials generate the data necessary to demonstrate the safety and efficacy of a product prior to its approval for use in humans.

CTM CRC researchers are developing two animal models for preclinical testing. The first is to test cell delivery devices for the treatment of chronic wounds. The second model allows for simulation of the human immune system, to investigate its response to foreign materials like cells and devices. A working model has been established and is currently being used in the development of islet transport devices.

Establishment of scale-up facility A facility for process development and manufacture of cell therapies was setup by NextCell Pty Ltd to assist with the translation of technologies into a manufacturing environment. In addition to small-scale manufacture of cell-based therapies, the facility will be used for the training of SME staff in cGMP manufacturing principles. This will address the skills shortage faced by the cell therapy industry in Australia and abroad. Training courses will provide hands-on training and experience to complement theoretical cGMP training.

Alignment of projects with regulatory requirements CTM CRC’s regulatory review of projects is an ongoing activity that continues to provide guidance to researchers on therapeutic product development. Involving experienced staff from CTM CRC participants, NextCell Pty Ltd and Cell Therapies Pty Ltd, the review considers the regulatory framework from all major jurisdictions, leading to strategic activities to address risks and challenges.

A knowledge management system continues to be used to manage CTM CRC project data. All electronic files and data related to CTM CRC projects are centralised in a secure electronic storage facility, which is backed up on a daily basis.

Advanced cell-based wound therapies

n Over 400,000 chronic wounds at any given time in Australia alone

n A collaboration between CTM CRC’s research and industry participants is developing a novel wound dressing to deliver therapeutic cells into wounds to speed up healing

n The dressing could negate the need for cell injections, which is particularly impractical for painful, difficult to heal wounds

n Results from preclinical testing in models of chronic wounds indicate faster healing, and with fewer cells than expected

n Planning is underway for the next phase of development, involving clinical trials in patients with chronic wounds

n The impact of this technology will be to reduce the cost and improve the clinical handling of such therapies, making them more affordable and accessible to the large number of chronic wound sufferers Dr Sue Low in CTM CRC’s scale-up facility

Research

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CTM CRC’s education and training programme aims to advance the emerging cell therapy industry in Australia by cultivating a workforce with the necessary skills.ePhD Programme Through CTM CRC’s flagship ePhD Programme, graduates acquire skills that equip them for a broad range of careers in the biomedical sector.

The training focuses on creating an industry-ready workforce with valuable transferrable skills and an understanding of research translation and commercialisation, thus instilling an entrepreneurial mindset.

Activities consist of four, intensive, in-residence sessions at CTM CRC, with a capstone project or industry placement in the final year. Students are also included in other CTM CRC-led professional and social opportunities to increase face-to-face engagement, enhance linkages to CTM CRC participants, and develop professional networks.

CTM CRC is on course to educate at least twenty PhD students through its ePhD Programme. To date, CTM CRC has awarded fifteen PhD and three Honours scholarships, developed the syllabus and delivered three, multi-day workshops for the ePhD Programme.

Student engagement CTM CRC enrolled four new PhD students, surpassing its student engagement milestones for the reporting period.

Two new PhD opportunities for students commencing in 2016 were created through the generous support of the Playford Memorial Trust. CTM CRC/Playford Trust PhD scholarships are awarded to high-achieving scholars who are committed to using their skills and research for the future development of industry in South Australia. Ms Anouck Burzava and Sk. Al Zaheri Mahmud were the 2016 recipients, and were formally recognised at an awards ceremony held in April 2016.

CTM CRC continues its financial support of Honours students to create a feeder pathway into its ePhD Programme. CTM CRC awarded two honours scholarship packages during the reporting period, with additional support to attend CTM CRC events.

Events Startup BootcampCTM CRC’s inaugural Startup Bootcamp, part of its ePhD Programme, was held in October 2015. The three-day programme, attended by ten students, was focused on cultivating entrepreneurial and enterprising researchers. The hands-on approach - encouraging students to develop and pitch their own business ideas, which were then evaluated by a panel of judges on the final day - maximised student participation and learning. Multiple guest speakers with real-world experience as innovators and entrepreneurs were present to help connect students to the broader entrepreneurial community.

PhDs@workCTM CRC’s third ePhD module, PhDs@work, focused on transferrable business skills commonly sought by employers in new graduates. The module, held over four days in April 2016, was attended by fifteen students, including two from the Data to Decisions CRC.

Attendees participated in workshops on Leadership and Teamwork, Project Management, Financial Management, Personal Branding and Networking, and Career Planning. The following positive endorsement was received from all students:

n ‘The course increased my knowledge and understanding of business skills’

n ‘The course was relevant to my overall needs as a PhD student’

n ‘I learnt things that I can put into practice straight away’

n ‘I felt interested and engaged throughout the course’

ImpaCT WorkshopCTM CRC’s inaugural two-day ImpaCT Workshop was held in June 2016 and brought together students from Adelaide, Sydney and Brisbane. Third year students provided research updates, second year students delivered pitches modelled on the 3-Minute Thesis®, to hone communication skills. The standard of presentations was very impressive, with prizes awarded to the best speakers from each group.

Industry development Bridging the gap between industry and researchers, and identifying and addressing skills gaps in the emerging industry of cell therapies, are important facets of CTM CRC’s education programme.

CTM CRC has, in conjunction with SeerPharma Pty Ltd, developed several training modules in cell therapy development, manufacturing and regulation. The first

workshop comprising these modules will be delivered in Adelaide in September 2016, meeting Output Milestone 2.4.5. CTM CRC is now well positioned to meet ongoing milestones related to industry training.

Collectively, the ePhD and industry training programmes will foster the commercialisation and growth of Australia’s expanding biomedical and cell therapy industries.

External engagement The education programme regularly engages with a wider audience through events and community engagement. During the reporting period, CTM CRC’s Education & Training Manager, Dr Andrew Milligan, presented a lecture in May 2016 to first year undergraduates at the University of Adelaide about the biotechnology industry, CTM CRC and cell therapies. He also presented a lecture on start-up biotechnology companies to students enrolled on the BBiotech degree at Flinders University, on 1 April 2016.

These external engagements increase the visibility of CTM CRC, and help promote pathways and career opportunities in the biomedical and STEM sectors, particularly where they interface with industry.

Student awards Burzava A – PhD ScholarshipPlayford Memorial Trust

Mahmud AZ – PhD ScholarshipPlayford Memorial Trust

Stead S - Mentor Mentee Travel AwardThe Transplant Society

Tan L – Finalist, Three Minute Thesis competitionUniversity of South Australia

Tan L – Australasian Society for Immunology Student Travel BursaryAustralasian Society for Immunology

Thompson E – Best Presentation AwardUniversity of South Australia 3rd Bi-Annual School of Pharmacy and Medical Sciences Symposium

Thompson E – Best Student Poster7th Barossa Meeting - Cell Signalling in Cancer Biology and Therapy

WW

Cell Therapy Manufacturing

Cooperative Research Centre

Cell Therapies Development, Regulation and ManufacturingFull Day WorkshopTuesday 27 September 2016 BioSA Conference Centre40-46 West Thebarton Road, Thebarton SA 5031

$220.00 (inc. GST)

Education & Training

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CTM CRC PhD students, Hannah Thomas & Hanieh Shirazi during CTM CRC’s Startup! Bootcamp

Student Commencement Date

Research Programme Project Title University Country Expected

Completion Date

Kristen Malatesta 26 Aug 2013 1 - Materials & Bioprocessing

The functional role of microRNA in human regulatory T cell subsets

Adelaide Australia Feb 2017

Lih Tan 10 Feb 2014 1 - Materials & Bioprocessing

Vasculogenic mimicry: regulation and function in melanoma

UniSA Australia Feb 2018

Adel Dalilottojari 17 Feb 2014 1 - Materials & Bioprocessing

Using high throughput screening to design cell therapy scaffolds

UniSA Australia Apr 2017

Lewis Martin 3 Mar 2014 1 - Materials & Bioprocessing

Optimising the attachment and orientation of peptides on polymers by stimulation and experiment

Sydney Australia Mar 2017

Emma Thompson 17 Mar 2014 1 - Materials & Bioprocessing

The potential role of interleukin-3 (IL-3) in blood vessel development in breast cancer

UniSA Australia Sep 2017

Hanieh Shirazi 30 June 2014 1 - Materials & Bioprocessing

Development of novel plasma polymer surfaces for adhesion, proliferation and harvesting of stem cells

UniSA Australia Jun 2017

Felix Wunner 30 Jul 2014 2 - Clinical Translation

Development of a technology platform for melt electrospinning

QUT Australia Oct 2017

Jazmin Ozsvar 19 Sept 2014 2 - Clinical Translation Elastic tissue repair Sydney Australia Jul 2017

Sebastian Stead 2 Feb 2015 2 - Clinical Translation

Dendritic cell targeting therapy utilising porous silicon nanoparticles

Adelaide Australia Feb 2018

Hannah Thomas 1 Mar 2015 2 - Clinical Translation

Flightless I regulation of pericyte function in diabetic chronic wounds

UniSA Australia Mar 2018

Gink Yang 1 May 2015 2 - Clinical Translation

Development of novel stem cell therapy for epidermolysis bullosa

UniSA Australia May 2018

Anouck Burzava 4 Jan 2016 2 - Clinical Translation

Development of a next generation arterial stent: a low fouling and endothelial progenitor cell selective platform

UniSA Australia Jan 2019

Sk Al Zaheri Mahmud 19 Jan 2016 2 - Clinical

Translation

Development of nanophotonic biosensor for mycoplasma detection

UniSA Australia Jan 2019

Argha Chakraborty 22 Feb 2016 2 - Clinical

Translation

Smart nanowire surfaces to generate CAR-T cells for melanoma treatment

UniSA Australia Feb 2019

Education & Training

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CTM CRC has a strong focus on translation, scale-up and commercialisation of its technologies. SME engagement, which provides invaluable input into several aspects of process and product development, is crucial to CTM CRC’s success. For example:

n CTM CRC continues to seek industry guidance and feedback for all its projects, ensuring that technology development remains relevant and current. Through their involvement with projects, industry end users provide CTM CRC with useful assessment and evaluation on practical, commercialisation and market feasibility. This, in turn, determines project outputs that provide value to industry and address end-user challenges.

n CTM CRC engages with SMEs at appropriate intervals within a project’s life cycle, consolidating project input with outcomes, outputs and utilisation pathways.

n CTM CRC’s approach to engage with SMEs early, and invite constant feedback, provides an ideal platform for collaboration. The focus on addressing specific ‘problem statements’ provides an ideal platform for meaningful CTM CRC/SME engagement and positions SMEs to build their capacity for innovation and growth.

SME participants are invited to participate in CTM CRC-wide activities such as the ePhD Programme. This ensures SME workforce needs are addressed through CTM CRC education and training packages.

CTM CRC has engaged directly with Australian cell therapy related SMEs through its research activities and is continuing to broaden its engagement with SMEs in allied fields. For example, CTM CRC is in discussions with SMEs that could provide tools and manufacturing capacity to add to CTM CRC’s portfolio of capabilities. This also has potential to contribute to the overall cell therapy manufacturing supply chain within Australia.

CTM CRC maintains active engagement with all its SME and industry participants through regular project meetings, ImpaCT workshops and internal communication channels.

“The coverage of cell therapies and the cGMP and clinical trial process was most valuable”Delegate response to CTM CRC’s ‘Cell Therapy Development, Regulation and Manufacturing’ workshop delivered by SeerPharma

Members of industry & academia at CTM CRC co-hosted ISCT ANZ Regional Meeting - ‘Translating Cell Therapy 2015’

SME Engagement

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OverviewAs detailed below, during the reporting period, CTM CRC undertook substantial utilisation and commercialisation activities including:

n Three new Australian provisional patent applications filed

n CTM CRC’s first International PCT patent application filed

n CTM CRC’s first spin-out company, Carina Biotech Pty Ltd, incorporated

Patent filingsDetails for the three new provisional patent applications are shown in CTM CRC’s IP portfolio (see table). These provisional patent applications related to: (i) A technology for the expansion of therapeutic T cells(ii) A new CAR-T cell which may have utility in the treatment of cancers(iii) An antifouling and antithrombotic stent comprising a chemically tailored surface coating

It should be noted that (i) and (ii) above relate to technologies developed in CTM CRC’s emerging immunotherapies theme and these patent applications may become part of Carina Biotech’s IP portfolio.

CTM CRC’s first international PCT patent application (see table) claims priority to a provisional application filed in the previous reporting period. The PCT builds on CTM CRC’s IP protection around its methods and products for delivering therapeutic cells to biological sites, such as chronic wounds. Athersys, Inc., has been appointed as a provisional utilisation party for this technology and a formal commercial IP transfer agreement on this technology is expected to be finalised during the next reporting period.

During the reporting period, CTM CRC incorporated its first spin-out company, Carina Biotech Pty Ltd (ACN 613 276 074).

Carina will seek to exploit commercial opportunities in the T cell immunotherapy industry, particularly in relation to CAR-T cells, which have been showing unprecedented results for the treatment of leukaemia in clinical trials. Carina plans to commercialise a suite of CTM CRC technologies around T cell ‘manufacture’ – particularly producing, growing and delivering T cells to patients. It is anticipated that these technologies will help improve the affordability and accessibility of T cell immunotherapies.

In addition, CAR-T cells can only kill a cancer cell when they have a suitable target on the cancer cell that they can ‘lock on’ to. The target for leukaemia is well known, but the next frontier for CAR-T cells is identifying suitable targets on other cancers, especially solid tumours. At Carina, work has also started on a new CAR-T cell that may target and kill these types of cancers.

Carina is wholly owned by CTM@CRC Ltd., although it is anticipated that further equity investment will be sought for the company in the coming years.

Utilisation milestonesDuring the reporting period, CTM CRC had four utilisation milestones, each related to the filing of a patent application. While, in some cases, the subject matter of the patent application was not exactly as projected in the milestone, CTM CRC filed four new patent applications, including three new Australian provisional patent applications and one new International PCT patent application.

Intellectual property management CTM CRC has a strong focus on generating impact from its research activities. Developing a strong IP portfolio and the protection and commercialisation of CTM CRC’s IP remains a strategic priority.

CTM CRC’s general manager, Dr Justin Coombs, is a registered patent attorney with substantial experience in bioscience commercialisation. This allows CTM CRC to take a strategic approach to how IP is protected and transacted.

CTM CRC’s business model has been carefully structured from its inception to facilitate the exploitation of technology developed through its research programmes, and centres around promoting the transaction of CTM CRC project IP into the hands of end users.

The legal framework for handling IP within CTM CRC remains unchanged from the previous reporting period, and has been structured to optimise the transaction of IP to the end-user and maximise the end-user benefit of CTM CRC research. In particular:

n All CTM CRC participants have agreed to assign CTM CRC project IP to the management company, CTM@CRC Ltd

n Where possible CTM CRC appoints a Utilisation Party (likely to be an industry participant) at the outset of each project

n Transaction of project IP to each Utilisation Party is formalised via a Utilisation Plan, which sets out the terms and conditions for IP transaction, exploitation and returns

IP identification and capture A strategy is in place for the rapid identification of IP emerging from CTM CRC projects.

CTM CRC’s general manager and research & development manager each maintain regular contact with project teams for briefings on research outputs. This allows the swift identification of potential IP as it is generated. A substantial benefit of this strategy is that it shifts the responsibility for IP identification on to management personnel who have substantial industry experience in IP identification, protection and commercialisation.

Publication guidelines also require CTM CRC researchers to submit any project-related publication to management for review prior to publication. This provides an independent secondary mechanism to identify and capture CTM CRC IP prior to any public disclosure.

carina b i o t e c h

Dr Justin CoombsGeneral Manager & IP Counsel, CTM CRC

Utilisation & Commercialisation

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National principles of intellectual property management for publicly funded research CTM CRC’s IP management strategy has been developed and implemented in accordance with the National Principles of Intellectual Property Management for Publicly Funded Research. With particular reference to these principles:

n CTM CRC’s capture and exploitation of project IP is intended to provide benefit to Australia, particularly to provide new medical technologies to Australian patients (Principle 2(b))

n Ownership of all IP generated through CTM CRC collaborative projects will vest in CTM@CRC Ltd, and these ownership arrangements have been agreed to by all CTM CRC participants in the Participant Agreements (Principle 2(c))

n CTM CRC has a clear policy position regarding the exploitation of CTM CRC project IP (Principle 2(d))

n The operational aspects of CTM CRC’s IP policy address Principles 2(e) and 2(f), specifically: CTM CRC’s IP policies are reflected in and disseminated to staff through CTM CRC’s Intellectual Property, Confidentiality and Disclosure policy

n CTM CRC also has specific policies in place

relating to the retention of project data. These policies are documented and disseminated to researchers in CTM CRC’s Good Scientific Practice policy and Use of CTM CRC Lab Books policy

n CTM CRC researchers have access to the management team for a broad range of IP and commercialisation advice

n All individual CTM CRC researchers and students are required to personally execute a Deed Poll that, among other things, confirms CTM@CRC Ltd ownership of IP generated in CTM CRC funded projects

n CTM CRC’s Participants’ Agreements clearly define the basis for how any commercial returns would be divided among participants

n The agreements also include provisions to enable academic publication of CTM CRC research in a way that is compatible with protection of CTM CRC project IP

Professional advisors To assist with its commercialisation and utilisation activities during the reporting period, CTM CRC retained the services of a range of professional advisors including: Phillips Ormonde Fitzpatrick for intellectual property as well as FAL Lawyers for commercial legal matters.

CTM CRC’s Registered IP PortfolioType Number Title Applicant Inventors Filing Date Priority

DateTrade mark (AU) 1589377 CTM@CRC Ltd N/A 12-Nov-13 12-Nov-13

Patent - PCT application

PCT/AU2016/050101 METHODS AND PRODUCTS FOR DELIVERING CELLS

CTM@CRC Ltd. SMITH, Louise Elizabeth; MICHELMORE, Andrew Percival; KIRBY, Giles Thomas Sipho; COWIN, Allison June; MILLS, Stuart James; SHORT, Robert David

16-Feb-16 16-Feb-15

Patent - AU prov. 2015903495 PRODUCTS AND METHODS FOR ACTIVATING AND/OR EXPANDING T CELLS

CTM@CRC Ltd. not yet named 28-Aug-15 28-Aug-15

Patent - AU prov. 2015903719 CHIMERIC ANTIGEN RECEPTOR AND USES THEREOF

CTM@CRC Ltd. COOMBS, Justin Taylor; BARRY, Simon Charles; SADLON, Timothy John

11-Sep-15 11-Sep-15

Patent - AU prov. 2016901008 ANTI-FOULING MEDICAL DEVICES

CTM@CRC Ltd. VOELCKER, Nicolas Hans; MOORE, Eli; BONDER, Claudine Sharon

17-Mar-16 17-Mar-16

Utilisation & Commercialisation

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CommunicationsCTM CRC’s integrated marketing and communications strategy provides a framework for communicating objectives, activities, achievements and successes to internal and external audiences. The strategy focuses on raising the external profile of CTM CRC in the cell therapy industry, and increasing communication as well as collaboration within CTM CRC. The strategy involves a number of internal and external engagements conducted throughout the year, including:

n Publication of CTM CRC’s annual ImpaCT magazine

n Hosting CTM CRC’s ImpaCT Day and first ImpaCT Workshop

n Co-hosting the 2015 ISCT ANZ Regional Meeting

n The redevelopment and redesign of the CTM CRC website

Internal communications CTM CRC’s internal communications activities in this reporting period were focused on encouraging and supporting collaboration amongst participants, including researchers and students.

Internal stakeholders were engaged via communications, publications and activities that included ImpaCT Days, the ePhD Programme, participant meetings, research project management meetings, email campaigns and quarterly reports. These activities were also designed to strengthen the sense of community and cohesion within CTM CRC.

ImpaCT Workshop In addition to the continued ImpaCT Day series, CTM CRC held its first two-day ImpaCT Workshop in Melbourne.

The ImpaCT Workshop, sponsored by Phillips Ormonde Fitzpatrick, was held to foster cross fertilisation amongst CTM CRC participants, encourage the exchange of ideas, and continue to strengthen collaboration.

With 80 attendees, the workshop included presentations by researchers and students around CTM CRC’s renewed focus on its strategy and potential new projects and ideas. Feedback from CTM CRC’s Board of Directors was provided, as well as presentations from industry representatives including, Mr Bill Mercer, former Vice President – Terumo BCT, Dr Stephen Wilson, Chief Operating Officer – Scinogy and Dr Justin Dibbens, Patent and Trade Marks Attorney – Phillips Ormonde Fitzpatrick. The workshop was well received and generated discussions and collaboration, providing a foundation for project proposals in preparation for CTM CRC’s next round of projects.

External communications CTM CRC’s external communications strategy employs a diverse range of delivery platforms including the CTM CRC ImpaCT magazine, media releases, radio broadcasts, brochures and associated marketing publications, networking events, public lectures, conference presentations, sponsorship and the CTM CRC website and social media.

2015 International Society for Cellular Therapy (ISCT) ANZ Regional Meeting CTM CRC co-hosted the 2015 ISCT ANZ Regional Meeting – ‘Translating Cell Therapy’. The conference was held at the University of South Australia and attracted 121 delegates, including members of national and international industry, as well as representatives from government and academia.

The conference was opened by Dr Sherry Kothari, CEO, CTM CRC and Dr Robert Nordon, former Regional Vice President, ISCT. The conference programme included over 24 presentations across three individual sessions, including a keynote presentation by Dr Shelly Heimfeld, Director - Heimfeld Laboratory, Fred Hutchinson Cancer Research Centre, Seattle, USA, and presentations by Dr Sherry Kothari and three CTM CRC researchers (Professor Simon Barry, Professor Rob Short and Dr Louise Smith).

Website and social media Website and social media are an integral part of CTM CRC’s internal and external communications strategies. The website, located at www.ctmcrc.com, hosts details about CTM CRC research and education programmes, participants, news, events and publications. It serves as an up-to-date portal of information for CTM CRC and its stakeholders.

CTM CRC also has social media accounts with Facebook, Twitter and LinkedIn. Social media is used by CTM CRC to distribute news and achievements

from CTM CRC and the cell therapy industry, as well as promote CTM CRC’s programmes, researchers and students. Social media aids in unifying and strengthening CTM CRC’s message to stakeholders online and drives traffic to the website.

Measurement of activity effectiveness CTM CRC measures its effectiveness through mechanisms such as its Participant Listening Programme, social media monitoring, website engagement statistics and direct feedback from its stakeholders and the community to determine the effectiveness of its communications strategies and to inform the development of future strategies.

CTM CRC’s 2016 ImpaCT Workshophosted by Phillips Ormonde Fitzpatrick in Melbourne

CTM CRC’s A/Professor Simon Barry presenting at 2015 International Society for Cellular Therapy (ISCT) ANZ Regional Meeting

Marketing & Communications

Social Media FollowingPlatform Followers % Change since

FY 14-15LinkedIn 223 ~32.7

Twitter 175 ~47.1

Facebook 82 ~60.8

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Event Highlights2 July, 2015 – ‘CTM CRC’s Birthday Bash’The Gallery, Adelaide

16 October, 2015 – ‘CTM CRC ImpaCT Day 2015 – Dragon’s Den’The Mayfair Hotel, Adelaide

16-17 October, 2015 – ‘Translating Cell Therapy 2015 – ISCT ANZ Regional Meeting’University of South Australia City West Campus, Adelaide

19-21 October, 2015 – ‘ePhD Module 2: Startup! Bootcamp’Innovation and Collaboration Centre, UniSA City West Campus, Adelaide

14 December, 2015 – ‘Regenerate yourself – the future of 3D bioprinting and organ transplantationSouth Australian Health and Medical Research Institute (SAHMRI), Adelaide

19-22 April, 2016 – ‘ePhD Module 3: PhDs @ work’University of South Australia City West Campus, Adelaide

31 May, 2016 - ‘Overview of Intellectual Property’The Space Environment Research Centre, Canberra

7-8 June, 2016 – ‘CTM CRC ImpaCT Workshop 2016’Phillips Ormonde Fitzpatrick, Melbourne

Sponsorship25-27 January, 2016 – ‘Phacilitate Cell & Gene Therapy World Conference’Hilton Alexandria, Washington DC

18-20 May, 2016 – ‘World Stem Cells & Regenerative Medicine Congress’Business Design Centre, London UK

Media Releases & PR11 August, 2015 – ‘CTM CRC, GE Healthcare and Health Industries SA sign strategic cooperation agreement to advance Australian cell therapy’CTM CRC Media Release

11 August, 2015 – ‘GE Healthcare joins forces with the Cell Therapy Manufacturing CRC and Health Industries South Australia’The Advertiser Business Journal, Adelaideadelaidenow.com.au

24 August, 2015 – ‘Strategic Agreement for advancing Australia’s cell therapy industry’Austrade.gov.au

12 December, 2015 – ‘Building a hi-tech state’The Advertiser Business Daily, Adelaide

14 December, 2015 – ‘CTM CRC hails new 3D printing capability developed by its latest participant, the University of Wollongong’CTM CRC Media Release

14 December, 2015 - ‘3D Printing and the treatment of diabetes’Fiveaa (radio interview)

14 December, 2015 – ‘Wound CRC and CTM CRC: Together Fostering Enterprising PhD Students’Wound Management Innovation CRC Newsletter - Edition 29

7 April, 2016 – ‘CTM CRC and the Women’s and Children’s Hospital launch a new project to advance cancer immunotherapy’CTM CRC Media Release

7 April, 2016 – ‘New cancer research project at WCH with Cooperative Research Centre for Cell Therapy Manufacturing’The Advertiser Business Journal, Adelaideadelaidenow.com.au

27 April, 2016 - ‘CTM CRC ImpaCT Magazine’CTM CRC Publication

25 May, 2016 – ‘UK’s Cell and Gene Therapy Catapult and the Australian CRC for Cell Therapy Manufacturing sign new agreement to advance scaffold technology for commercial production of T cells’CTM CRC Media Release

26 May, 2016 – ‘UK, Australia research institutes collaborate on T cell stimulation technology’Biospectrum

8 June, 2016 – ‘New treatments for diabetes from emerging technology to be presented at SAHMRI’adelaidenow.com.au

Dr Dominic Wall and attendees at CTM CRC’s 2015 ImpaCT Day - ‘Dragon’s Den’ at the Mayfair Hotel, Adelaide

Marketing & Communications

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CTM CRC StructureCTM CRC is managed by a not-for-profit company limited by guarantee, CTM@CRC Ltd. The company has tax-exempt status, and endorsement from the Australian Charities and Not-for-profits Commission (ACNC) and the Australian Taxation Office (ATO) as a health promotion charity. The company operates a lean management structure to maximise its spend on research activities, and has overall responsibility for the delivery of the agreed research outputs, utilisation and impacts of CTM CRC.

CTM CRC participants comprise companies in the cell therapy industry, research institutions, universities and hospitals. Their involvement in CTM CRC is governed by an Participants’ Agreement.

CTM CRC BoardCTM@CRC Ltd is governed by an independent, skills-based board of directors. The Board’s main responsibility is to provide high-level governance for CTM CRC, as well as financial accountability and oversight of compliance with Commonwealth and other appropriate laws and regulations.

In addition to monitoring CTM CRC performance, the Board plays an important role in the approval and regular review of all projects to ensure their alignment with CTM CRC objectives.The Board maintains oversight of CTM CRC finances to ensure compliance with the Australian Securities and Investments Commission (ASIC) and ACNC regulations and to ensure that funds are expended appropriately.

CTM CRC’s six directors, including its Managing Director are each appointed for a period of three years.Both the chair and directors can be re-elected by a special majority vote (75%) of members. Essential Participants of CTM CRC, who are members and are entitled to vote at company general meetings.

There was no change to the board of directors during the reporting period.

Board MembersLeanna Read, PhD, FAICD, FTSEDr Leanna Read has extensive executive, board and investment experience in technology-based enterprises. She has a unique track record that spans the public and private sectors, particularly in biotechnology.

Dr Read has extensive experience in the CRC sector, including previous roles as CEO of the CRC for Tissue Growth and Repair, and a board member of two other CRCs. In 2001, Dr Read founded South Australian biotechnology company, TGR BioSciences Pty Ltd (a CRC spinout), and served as the company’s Managing Director and CEO until 2012. She has also served on the Prime Minister’s Science, Engineering and Innovation Council, the Federal Government’s IR&D Board and the Commercialisation Australia Board.

Dr Read holds the position of Chief Scientist for South Australia. She is also currently a member of boards that address innovation across the government, industry and academic sectors, including the Federal Government Biomedical Translation Fund Committee, the South Australian Economic Development Board, the South Australian Science Council and the University of South Australia Council. She is a fellow of the Australian Institute of Company Directors and of the Australian Academy of Technological Sciences and Engineering. Dr Read has been the recipient of a number of prestigious local and national awards.

Sherry Kothari, PhD, MBA, GAICDDr Sherry Kothari has been a founder-investor in two successful life sciences spin-out companies and brings extensive experience in working within bioscience at the research/industry/clinical interface.

Dr Kothari started her career in maxillofacial surgery before undertaking a PhD in biomaterials and tissue engineering, followed by an MBA. As well as her interest and experience in medicine and the cell-materials interface, Dr Kothari has a passion for entrepreneurship and translation within the biotechnology sector.

With a strong focus in strategic leadership, organisational culture and communication, Dr Kothari excels in working at cross-disciplinary boundaries to harness expertise within multidisciplinary teams to deliver tangible commercial outputs. Dr Kothari has established strategic partnerships across Australia, Asia, Europe and North America.

Dr Kothari is a recipient of the Women In Innovation SA Award (2014) for Science and Health and is a graduate of the Australian Institute of Company Directors. Dr Kothari is also a member of the Regenerative Medicine Advisory Group for Ausbiotech, Australia’s biotechnology organisation, with a network of over 3,000 members in the life sciences.

CTM CRC’s Board of Directors(L-R) Dr Leanna Read, Dr Stephen Livesey, Mr Ray Wood, Dr Alexander Gosling, Dr Sherry Kothari, Mr Charilie Latham

CTM CRC’s Chair, Dr Leanna Read, presenting at the 2016 ImpaCT Workshop

Governance

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Stephen Livesey, MBBS, PhDDr Stephen Livesey has a career history spanning medicine, medical research and commercialisation.

After graduating from his medical and postdoctoral studies at the University of Melbourne in 1985, he has held a number of positions in Australia and the US. In 1986, Dr Livesey co-founded LifeCell Corporation in the US, through a technology transfer from the University of Texas. The company was established to commercialise a cell and tissue preservation technology, which he invented (now marketed as AlloDerm™).

Following a period in Australia as a Wellcome Trust Senior Research Fellow, Dr Livesey returned to the US to become Executive Vice President and Chief Science Officer of LifeCell. He was the lead scientist in securing additional funding for LifeCell, including venture capital, an initial public offering on the NASDAQ, private placements, follow-on offerings and public offerings, totaling approximately US$78 million. AlloDerm has been used in more than two million surgical procedures with more than 600 peer-reviewed publications on its clinical and research use. In 2007, LifeCell Corporationwas acquired by Kinetic Concepts International (NYSE:KCI) for US$1.7 billion.

In 2003, Dr Livesey returned to Australia and joined the Australian Stem Cell Centre, where he served as the Chief Scientific Officer and CEO. He maintains an active research interest in matrix-based research for spinal cord injuries, bone marrow transplantation and cartilage repair at St Vincent’s Hospital, Melbourne.

Alexander Gosling AM, DEng, FTSE, FIEAustDr Alexander Gosling has been working in the field of process and product development for 40 years.

Dr Gosling holds a Masters in Engineering from the University of Cambridge and an honorary doctorate from Swinburne University of Technology. As a founding director of Invetech, an engineering and process development consultancy, he has serviced clients ranging from high tech start-ups to ‘smoke-stack industry’ global companies. He was part of the management team that led Invetech firstly to a public listing (as Vision Systems Ltd), and then to its acquisition by the US Danaher group for over $800 million.

Dr Gosling is a past national president of the Australasian Industrial Research Group, a past national president of the Australia-Malaysia Business Council, a Fellow of the Academy of Technology and Engineering,

a Fellow of the Institute of Engineers Australia, and a Governor of the Warren Centre for Advanced Engineering. He also sits on a number of industry advisory committees. Dr Gosling has been appointed a Member of the Order of Australia for services to business through innovative support for research and development, and to the community.

Currently, Dr Gosling is a Director on the Boards of the Innovative Manufacturing Cooperative Research Centre, Micro-X Ltd (an Adelaide-based listed company manufacturing a novel mobile medical X-ray system) and Bush Heritage Australia.

Charlie Latham, CA, GAICD, FCA (England)Mr Charlie Latham is a Chartered Accountant with a background in science, finance and compliance. He has over 40 years’ professional experience across a broad range of industries including life sciences, manufacturing, engineering, investment and service industries.

Mr Latham began his career with an Honours degree in zoology, before transitioning to accounting, finance and auditing with professional accounting firms, including Deloitte. He has had over 20 years’ board involvement as Company Secretary and Chief Financial Officer (CFO) of small ASX-listed entities as well as extensive experience as a director, CFO and secretary of other public and private companies. He has served on a number of audit and risk committees, due diligence committees and capital raising committees, and completed numerous acquisitions and disposals of businesses.

Mr Latham has strong professional and government links in South Australia, and extensive networks within the Australian bioscience industry.

Ray Wood, FAICDMr Ray Wood has more than 35 years’ experience in developing and deploying Australian innovation and technology into global markets.

Mr Wood holds a BSc(Tech) Electronic Engineering. His experience ranges from the continuous flow pathology analysers of the early 1970s and the first automated haematology counters, through to the development and marketing of diagnostic ultrasound machines, and, more recently, delivering professional consulting services and creating biotechnology spin outs.

Mr Wood has held roles within a range of organisations, including Plessy Telecommunications, Technicon Inc, Ausonics, PA Technology, Invetech, X-ray Technologies

and he was a founding stakeholder and Managing Director of Cell Therapies Pty Ltd. He has significant experience in the emerging field of cellular therapies and regenerative medicine. This includes the critical integration of the cGMP code and regulatory oversight.

Mr Wood is a non-executive director of CTM CRC. He also holds a non-executive Director position with the Victorian Clean Technology Fund and is a director and principal of Irendos Pty Ltd. He is a Fellow of the Australian Institute of Company Directors and a retired Fellow of the Institute of Engineers Australia.

Board meetings & attendanceDuring the reporting period board meetings were held in September, December, February, March and June. Attendances are documented in the following table:

9 & 10 Sep

3 & 4 Dec

21 Mar 8 & 9 Jun

Dr Leanna Read YES YES YES YES

Dr Sherry Kothari YES YES YES YES

Mr Charlie Latham YES YES YES YES

Dr Alexander Gosling NO YES YES YES

Dr Stephen Livesey YES YES YES YES

Mr Ray Wood YES YES YES YES

Board Member Key SkillsName Role Key Skills Independent/

OrganisationDr Leanna Read Chair Industry; research management; corporate governance Independent

Dr Sherry Kothari Chief Executive Officer and Managing Director Research/industry/clinical interface; cell therapy commercialisation CTM@CRC Ltd

Mr Charlie Latham Independent Director; Company Secretary Financial management; corporate governance Independent

Dr Alexander Gosling Independent Director Technology commercialisation; manufacturing process development Independent

Dr Stephen Livesey Independent Director Cell therapy commercialisation; technical expertise; regulatory expertise Independent

Mr Ray Wood Independent Director Cell therapy manufacture and commercialisation; regulatory expertise Independent

Governance

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The primary function of the Audit and Risk committee is to support the board in exercising due care and diligence. It assists the Board in discharging its oversight and monitoring responsibilities in relation to risk management, internal and external audit, financial statements and other matters at the Board’s request.

The committee meets at least four times per year, and has a number of responsibilities, including:

n Providing open lines of communication between CTM CRC’s external auditors and the Board

n Overseeing processes that identify and assess and mitigate general business risk, and advising the Board as necessary

n Reviewing the adequacy of CTM CRC’s internal control systems with management and external auditors

n Reviewing any significant risks, findings and recommendations made by external or internal auditors

Suzanne Ridding, MBAMs Suzanne Ridding is an experienced sustainability consultant, finance and corporate services executive, banker, board director and company secretary. She has held leadership positions in private, public and not-for-profit sector organisations internationally and in Australia. Ms Ridding has managed her own successful sustainability consultancy since 2008 specialising in organisation sustainability, carbon management and climate adaptation.

Ms Ridding was a non-executive director at the Risk Management Institution of Australasia for over three years. She has also worked extensively with boards and audit committees, including in her role as Chief Financial Officer and Company Secretary of TransAdelaide and as the Financial Controller at HomeStart Finance.

Ms Ridding’s key skills and experience include strategy and policy development, extensive risk management, corporate governance, financial management and sustainability.

CTM CRC’s Audit & Risk Committee(L-R) Mr Charlie Latham, Ms Suzanne Ridding, Mr Ray Wood

Dr Stephen Livesey at CTM CRC’s 2015 ImpaCT Day - ‘Dragon’s Den’ at the Mayfair Hotel, Adelaide

Audit and Risk CommitteeName Role Independent/

organisationMr Charlie Latham Chair CTM@CRC Ltd Board

Mr Ray Wood Member CTM@CRC Ltd Board

Ms Suzanne Ridding Member Independent

Governance

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CTM CRC Senior Management

General Manager & IP CounselJustin Coombs, MIPLaw, PhD, FIPTADr Justin Coombs has overall responsibility for the operations of CTM@CRC Ltd, as well as providing in house IP counsel to CTM CRC. in 2016 Dr Coombs was elected for entry into the Australian American Leadership Dialogue Young Leadership Dialogue.

Program Manager, R&DTony Simula, MBA, PhDDr Tony Simula manages and oversees CTM CRC’s research programme to ensure tangible outcomes and identify appropriate commercial opportunites.

Program Manager, EducationMonica Kerr, PhD (outgoing)Andrew Milligan, MBA, PhD (incoming)Dr Andrew Milligan oversees the design and execution of CTM CRC’s ePhD programme. Dr Milligan also has responsibility for the development of CTM CRC’s industry training programme.

CTM CRC CapabilitiesCTM CRC brings together a multidisciplinary team of biomaterials scientists, cellular biologists, clinicians, manufacturing and regulatory experts to co-develop technologies to improve current methods of cell therapy manufacture where there are significant impacts on the cost of goods.

CTM CRC’s capabilities extend across its three research themes and encompass:

n Materials development and advanced surface coatings

n Surface analytical techniques

n Cell biology of various therapeutic cells

n Process scale-up

n Access to expert clinicians utilising cell therapies

n Preclinical evaluation of biomedical devices and therapies

n Bioanalytical and product potency testing

n Regulatory affairs

“By leveraging our combined international networks and expertise, we are ensuring that our endeavours in this promising field are as efficient and effective as possible, bringing cell therapies to market faster.”Dr Sherry Kothari, CEO, CTM CRC

Together, CTM CRC’s participants contribute 30.6 FTEs towards research and 12.7 FTEs in technical capacities.

CTM CRC Team(L-R) Ms Paula Borlase, Ms Katrina van Zanten, Dr Andrew Milligan, Dr Sue Low, Ms Natalie Watkins, Dr Sherry Kothari, Dr Justin Coombs, Dr Tony Simula

Key Staff

Name Organisation CRC position/role Time committed (FTE)

Dr Sherry Kothari CRC for Cell Therapy Manufacturing CEO & Managing Director 1

Dr Justin Coombs CRC for Cell Therapy Manufacturing General Manager 0.9

Dr Tony Simula CRC for Cell Therapy Manufacturing Program Manager, R&D 1

Dr Monica Kerr CRC for Cell Therapy Manufacturing Program Manager, Education (outgoing) 1

Dr Andrew Milligan CRC for Cell Therapy Manufacturing

Program Manager, Education & Training (incoming) 1

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AthersysJef PinxterenAnthony TingLiesbeth Vandenpoel

Cell TherapiesTim OldhamJeanette RipperJustin VerwoertDominic Wall

NextCellDijana JerkovicSue LowGordon McPhee

Queensland University of TechnologyKenneth BeagleyDietmar HutmacherElena Juan-PardoDaniella Loessner

Royal Adelaide HospitalGlen BenvenisteToby CoatesChris DrogemullerJodie NitschkeDaniella PenkoDarling Rojas-Canales

Royal Prince Alfred HospitalJanet MacphersonJohn Rasko

St Vincent’s Institute of Medical ResearchGaurang JhalaCameron KosTom LoudovarisLina MariannaChristina TanHelen ThomasMichaela Waibel

SA PathologyClaudine BonderMichaelia CockshellLisa EbertSamantha EscarbeAngel LopezEli MooreKate Parham

Terumo BCTMichael KinzieKim Nguyen

The University of AdelaideVeronika BandaraCheryl Brown Batjargal Gundsambuu

University of South AustraliaSameer Al-BatainehAnton BlencoweKyle BrewerAnouck BurzavaAllison CowinBahman DelalatAurelien ForgetSoraya Rasi Ghaemi Hans GriesserFran HardingBenjamin Rink HofmaMarek JasieniakGiles KirbySteven McInnesAndrew MichelmoreStuart MillsNeethu NinanNick RogersRob ShortLouise SmithTong TongKrasimir VasilevNico VoelckerJason Whittle

The University of SydneyMarcela BilekAlexey KondyurinElena KosobrodovaDavid McKenzieTony WeissGiselle Yeo

University of WollongongGordon WallaceZhilian Yue

Women’s and Children’s Health NetworkSimon BarryTim Sadlon

CTM CRC Research & Manufacturing Staff

“CTM CRC provides participants with a great environment for doing exciting collaborative research. The focus on industry is really worthwhile and helps us get exciting outcomes.”Prof Anthony Weiss, The University of Sydney

Materials & Interfaces

Developing cheaper and more practical cell isolation, expansion and delivery technologies through the use of smart materials/lattices

and functionalised surfaces

Cellular Immunotherapies

Addressing the efficacy, manufacturing and delivery

challenges facing the T cell therapy industry

Pilot Manufacturing

Scale-up of surface-functionalisation and cell

expansion capabilities for proof-of-concept studies and rapid

translation of technologies

Key Staff

CRC for Cell Therapy Manufacturing Annual Report 2016 39

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During the reporting period, the University of Wollongong joined CTM CRC as an Other Participant. “CTM CRC presents a

potential commercialisation pathway as our technology develops”Prof Gordon Wallace, University of Wollongong

Participant Participant Type Australian Business Number Organisation Type

Athersys, Inc. Essential Participant International Industry

Cell Therapies Pty Ltd Essential Participant 15 100 285 916 Industry

Exylika Pty Ltd Essential Participant 45 158 849 980 Industry

MedVet Science Pty Ltd Essential Participant 15 008 089 745 Industry

NextCell Pty Ltd Essential Participant 85 164 452 886 Industry

Queensland University of Technology Essential Participant 83 791 724 622 University

Royal Adelaide Hospital - a division of Central Adelaide Local Health Network Essential Participant 96 269 526 412 Hospital

Royal Prince Alfred Hospital (Sydney Local Health District) Other Participant 17 520 269 052 Hospital

St Vincent’s Institute of Medical Research Essential Participant 52 004 705 640 Research Institute

SA Pathology - a division of Central Adelaide Local Health Network Essential Participant 96 269 526 412 State Government

South Australian Health and Medical Research Institute Ltd Other Participant 54 141 228 346 Research Institute

Terumo BCT Australia Pty Ltd Essential Participant 87 130 046 865 Industry

University of South Australia Essential Participant 37 191 313 308 University

University of Sydney Essential Participant 15 211 513 464 University

University of Wollongong Other Participant 61 060 567 686 University

Women’s and Children’s Health Network Inc. Other Participant 64 021 748 126 Hospital

Participants

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The substantial progress made within CTM CRC’s first three years is testament to the collaborative spirit within CTM CRC and the cohesion between research teams. Most of CTM CRC’s project teams are both multidisciplinary and multiinstitutional. CTM CRC currently also collaborates with a number of external research organisations such as The University of Nottingham and UCL.

Effective engagement with industry, both within and outside CTM CRC, has resulted in tangible outcomes around product profiles and prototypes. As projects progress towards near-commercial goals, CTM CRC’s industry engagement strategy is proving successful, with testing and integration into existing commercial systems already underway for some projects. CTM CRC’s open and transparent approach and a strong awareness of translational and commercial issues continue to foster collaboration.

Working closely with industry is integral to the development of technologies that can be easily integrated into manufacturing processes to deliver efficiencies, cost-reduction and improvements for cell therapy manufacture.

CTM CRC has continued to build on the strategic collaborative alliances formed with international organisations such as CGT Catapult. Proof-of-concept data generated through CTM CRC’s R&D platform to address specific industry challenges is generating considerable interest. This has led to new collaborative relationships with industry, providing a useful conduit to utilisation and commercialisation of CTM CRC technologies.

To facilitate the translation of novel cell therapies, CTM CRC is establishing a pilot scale-up facility. This facility will be integral to validating laboratory scale technologies for scale-up feasibility. This is often the first ‘valley of death’ encountered in the pathway to commercialisation, providing a mechanism to successfully navigate this is a crucial step in ensuringsuccessful technology transfer and scale-up of promising technologies. CTM CRC will leverage its strategic collaborations with cell therapy tools companies to achieve this.

The facility will also be used as a training hub that will offer harmonised, hands-on cGMP training tailored to cell and gene therapy. CTM CRC is working closely with CGT Catapult on this and the first modules are likely to be ready for delivery in early 2017. This will ideally position CTM CRC to start addressing the current worldwide shortage of skilled personnel for this field.

CTM CRC’s Professor Gordon Wallace at ‘Regenerate yourself - the future of 3D bioprinting and organ transplantation’ at SAHMRI

Collaboration

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n CTM CRC retained the services of Lee Green & Co. to provide day-to-day accounting services and preparation of regular management accounts

n William Buck was retained to provide audit services and to assist with the preparation of the financial statements

n Overall, total expenses for CTM CRC were very close to budget (~5% under budget)

n All budgeted contributions from participants were received in this reporting period

n In addition, CTM CRC attracted significant additional income in this reporting period. Most significantly, CTM CRC received:

� Additional SA Government-derived grant funding ($800k)

� An Export Market Development Grant ($48k)

� Interest income ($28k)

n As a result of the slightly lower expenditure and significantly increased income, CTM CRC ended the reporting period with an accrued surplus of $3.65m, an increase of $1.19m relative to the end of the previous reporting period

n It is anticipated the accrued surplus (including additional grant income) will be spent down over the remaining funding term, particularly in later years where the level of Commonwealth funding is reduced relative to the current reporting period

n No significant issues with respect to financial management were experienced in this reporting period

Financial statementsFinancial statements for CTM CRC have been prepared in accordance with the Australian Accounting Standards and the Commonwealth Government reporting requirements for CRCs and can be made available on request.

Mr Henry Botha teaching finance to CTM CRC’s students at the PhDs@Work ePhD module

Financial Management

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Conference PresentationsBarry S. Treg cell therapy: Tools for isolation, biomarkers of stable function and affordable manufacturingRegional ISCT Meeting, Adelaide, October 2015

Kothari S. Cell therapy in AustraliaRegional ISCT Meeting, Adelaide, October 2015

Myo Min K, Parham K, Rojas-Canales D, Penko D, Drogemuller C, Coates PT and Bonder C. Revealing a critical cross-talk between beta islet cells and the vasculature in diabetes (poster)Joint IPITA-IXA-CTS (International pancreas and islet transplant association, International xenotransplantation association, Cell transplant society) Congress Nov 2015, Melbourne, Australia

Myo Min K, Parham K and Bonder C. Revealing a critical cross-talk between beta islet cells and the vasculature in diabetes21st annual student meeting of the Australasian Society for Immunology SA/NT branch 2015, Adelaide, Australia

Myo Min K, Parham K and Bonder C. Revealing a critical cross-talk between beta islet cells and the vasculature in diabetesAustralian Society for Medical Research (ASMR) SA Annual Scientific Meeting 2015, Adelaide, Australia

Penko D, Rojas-Canales D, Forget A, Harding F, Delalat B, Waibel M, Loudovaris T, Blencowe A, Drogemuller C, Voelcker NH, Coates PT. Covalently bound islet survival factors to tissue culture surfaces maintain their biological activityInternational Pancreas and Islet Transplantation Association Congress, Melbourne, Nov 2015

Short R, Opportunities for material science in cell therapy manufacturingRegional ISCT Meeting, Adelaide, October 2015

Tan L, Thompson E, Sheppard K, Mintoff C, Shackleton M, MacArthur G, Ruszkiewicz A, Brown M, Bonder C, Ebert L. A critical role for desmoglein-2 in melanoma vasculogenic mimicryBarossa Cell Signaling Meeting Nov 2015

Tan L, Thompson E, Sheppard K, Mintoff C, Shackleton M, MacArthur G, Ruszkiewicz A, Brown M, Bonder C, Ebert L. A critical role for desmoglein-2 in melanoma vasculogenic mimicryAdelaide Immunology Retreat (AIR-11) 2015

Thompson E, Khew-Goodall Y, Lindeman G, Lopez A and Bonder C. The potential role of interleukin-3 (IL-3) in blood vessel development in breast cancerBarossa Cell Signaling Meeting Nov 2015

Cockshell M P, Moldenhauera L, Dalilottojari A, Harding F, Voelcker N and Bonder C S. Rapid and large-scale expansion of human endothelial progenitor cells with therapeutic potentialISCT Conference, Singapore, May 2016

Harding F, Delalat B, Gumdsambuu B, Mohandas A, Hill D, Walsh J, Pederson S, Grose R, Krumbiegel D, Couper J, Brown C, Sadlon T, Hutmacher D, Voelcker NH, Barry SC. Scalable functionalised scaffolds for clinical expansion of human T cellsISCT Conference, Singapore, May 2016

Kothari S. Cell Therapies – Opportunities and challenges in AustraliaISCT Conference, Singapore, May 2016

Michelmore A. Plasma polymers for biomedical devices: Fabrication of stable functionalised surfacesGaseous Electronics Meeting, Geelong, February 2016

Simula A. A ‘material’ improvement to COGSCell & Gene Therapy World Conference, Washington DC, January 2016

Simula A. Improved COGS – A tale of three surfacesWorld Stem Cell & Regenerative Medicine Congress, London, May 2016

Tan L, Thompson E, Sheppard K, Mintoff C, Shackleton M, MacArthur G, Ruszkiewicz A, Brown M, Bonder C, Ebert L. A critical role for desmoglein-2 in melanoma vasculogenic mimicryMedical Research Week South Australian Scientific Meeting, ASMR, May 2016

Thompson E, Khew-Goodall Y, Lopez A and Bonder C. The potential role of interleukin-3 (IL-3) in blood vessel development in breast cancerASMR Annual Meeting SA Branch 2016, May 2016

Non-conference PresentationsTan L, Thompson E, Sheppard K, Mintoff C, Shackleton M, MacArthur G, Ruszkiewicz A, Brown M, Bonder C, Ebert L. A critical role for desmoglein-2 in melanoma vasculogenic mimicryAdelaide Protein Group Student Awards 2016

Tan L, When benign moles go badThree Minute Thesis Grand Final (University of South Australia), 2015

Thompson E, Khew-Goodall Y, Lopez A and Bonder C. The potential role of interleukin-3 (IL-3) in blood vessel development in breast cancerCentre for Cancer Biology internal seminar series, 2016

Thompson E, Khew-Goodall Y, Lopez A and Bonder C. A role for interleukin-3 (IL-3) in breast cancer progressionUniversity of South Australia 4th school of Pharmacy and Medical Sciences seminar

Publications & Journal ArticlesEbert LM, Tan LY, Johan Z, Myo Min K, Cockshell M, et al. A non-canonical role for desmoglein-2 in endothelial cells: Implications for neoangiogenesisAngiogenesis. (2016) doi: 10.1007/s10456-016-9520-y

Kosobrodova E, Kondyurin A, Chrzanowski W, McKenzie D, Bilek M. Plasma immersion ion implantation of a two-phase blend of polysulfone and polyvinylpyrrolidoneMaterials and Design, 97(2016): 381-391

Ryssy J, Prioste-Amaral E, Assuncao DFN, Rogers N, Kirby GTS, Smith LE, Michelmore A. Chemical and physical processes in the retention of functional groups in plasma polymers studied by plasma phase mass spectroscopyPhysical Chemistry Chemical Physics 2016, 18 (6), 4496-4504

Saboohi S, Al-Bataineh SA, Safizadeh Shirazi H, Michelmore A, Whittle JD. Continuous-Wave RF Plasma Polymerization of Furfuryl Methacrylate: Correlation Between Plasma and Surface ChemistryPlasma Processes Polym., 2016, in press DOI: 10.1002/ppap.201600054

Tan LY, Mintoff C, Johan ZM, Ebert BW. Et al. Desmoglein-2 is a key regulator of vasculogenic mimicry and associated with poor clinical outcome in human melanomaOncotarget (2016) Jun 22. doi: 10.18632/oncotarget.10216

CTM CRC researcher, Dr Nick Rogers, presenting at the 2016 ImpaCT Workshop

Publications & Presentations

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Acronym Description Acronym Description Acronym

3D Three dimensional FBSE Fellow of Biomaterials Sceince and Engineering NASDAQ National Association of Securities

Dealers Automated Quotations

ACNC Australian Charities and Not-for-profits Commission

FCA (England)

Fellow of the Institute of Chartered Accountants in England

PCT Patent Cooperation Treaty

ASIC Australian Securities and Investments Commission FIEAust Fellow of the Institution of

Engineers Australia PhD Doctor of Philosophy

ASMR Australian Society for Medical Research FIPTA

Fellow of the Institute of Patent and Trade Mark Attorneys of Australia

PSC Pluripotent Stem Cell

ATO Australian Taxation Office FRACI Fellow of the Royal Australian Chemical Institute Q&A Question and Answer

bn Billion FTSEFellow of the Australian Academy of Technological Sciences and Engineering

R&D Research and Development

CAMember of the Institute of Chartered Accountants in Australia

GAICDGraduate Member of the Australian Institute of Company Directors

SA South Australia

CAR-T Chimeric Antigen Receptor T cell IL-3 Interleukin-3 SAHMRI South Australian Health and Medical Research Institute

CEO Chief Executive Officer IP Intellectual Property SME Small and medium enterprises

CFO Chief Financial Officer IPITA International Pancreas and Islet Transplant Association STEM Science, Technoloygy,

Engineering and Mathematics

cGMP Current Good Manufacturing Practice IR&D Independent Research and

Development TERMISTissue Engineering and Regenerative Medicine International Society

CRC Cooperative Research Centre ISCT International Society for Cellular Therapy TGR Tissue Growth and Repair

CTM CRC Cooperative Research Centre for Cell Therapy Manufacturing IXA International Xenotransplantation

Association Tregs Regulatory T cells

CTS Cell Transplant Society M Million UCL University College London

DEng Doctor of Engineering MBA Master of Business Administration UK United Kingdom

EPC Endothelial Progenitor Cell MBBS Bachelor of Medicine, Bachelor of Surgery UniSA The University of South Australia

ePhD Entrepreneurial PhD MDQ Management Data Questionnaire US United States of America

FAICD Fellow of the Australian Institute of Company Directors MIPLaw Master of Intellectual Property

Law WCH Women’s and Children’s Hospital

Glossary of Terms

CRC for Cell Therapy Manufacturing Annual Report 2016 49

Level 5Catherine Helen Spence BldgUniSA City West CampusADELAIDE, South AustraliaAUSTRALIA 5000

+61 8 8302 3503

Cell Therapy Manufacturing

Cooperative Research Centre

ctmcrc.com