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ISCT ANZ 2025 Regional Meeting Recap

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Guarav Sutrave, MBBS, FRACP, FRCPA, PhD The Westmead Institute for Medical Research The University of Sydney Australia	Alicia Didsbury, PhD ISCT ESP Committee Member University of Auckland New Zealand
Ali Shokoohmand, PhD ISCT ESP Committee Member The University of Queensland Australia	Gemma Moir-Meyer, MSc, PhD ISCT ESP Committee Member University of Otago New Zealand
Jessica Sue, BSc, MHSM ISCT Telegraft Committee Member ISCT Lab Practices Committee Member Sydney Cord Blood Bank Sydney Children's Hospital Network Australia

The 2025 ISCT Australia and New Zealand (ANZ) Regional Meeting was again bursting at the seams as delegates were welcomed to Manly – a beautiful beachside suburb in Sydney, Australia. Sold out for the second year running, attendees joined in from across Australia and Aotearoa, but also from as far away as Southeast Asia, India, South Africa and the United States. The 3-day scientific program (13-15 August) was preceded by a 2-day manufacturing workshop (11-12 August, which was also sold out despite having triple the capacity of our 2024 workshop) in partnership with Miltenyi Biotec, Merck Life Science, Thermo Fisher Scientific and Terumo Blood and Cell Technologies.

1. Winter sunshine streaming into the conference room early on Wednesday morning.

Day One began with ANZ Regional Vice President (and Sydney local) A/Prof. Emily Blyth (Westmead Institute of Medical Research; Westmead Hospital) opening the meeting and inviting Uncle Michael West to say Bujari Gamarruwa (Good Day). Michael led delegates on a historical tour of Sydney and shared the story of how Manly cove was named after the Cannalgal and Kayimai clan members who made a lasting first-impression as strong, confident and “manly” as they waded through the surf.

2. Fellow scientist, Uncle Michael West, a member of the Stolen Generations and
Aboriginal man of the Gamilaroi Nation, welcoming delegates to Manly, Sydney.

Manly’s beautiful beach views were popular with the international conference delegates, including ISCT President Elect Dan Weiss (University of Vermont), who managed to spend Tuesday afternoon exploring the area before taking to the stage for his address, “ISCT: Our legacy of purpose”. Dan’s talk highlighted the organisation’s distinctive role as a truly translational cell and gene therapy CGT society, driven by a mission to deliver CGT to patients across the world. To do so, ISCT acts as a facilitator to multiple different stakeholders, bringing together scientists, clinicians, regulators, and industry professionals. Almost a quarter of the ISCT community, who number more than 4,000 globally, hail to the Asia Pacific region (24%), with ANZ comprising 9% of all membership.

3. ISCT Global President Elect, Professor Dan Weiss, presenting "ISCT: Our legacy of purpose."

ISCT Institute of Training & Development is a strong focus within ISCT in recognition of the importance of training early career professionals (ESPs) for the development and delivery of these advanced, but also relatively nascent, therapeutics. In fact, ISCT is the only CGT society providing nationally accredited (ANAB – ANSI National Accreditation Board) workforce development courses. It was fitting then, that the first scientific session of the conference featured three ESP presenters whose work had been selected from a record number (70) of abstracts submitted to the conference.

Dr. Peter Eggenhuizen (Monash University): regulatory T cells induced with food antigen specific T Cell Receptor suppress IBD-related inflammation and colon damage in IBD mice exposed to the food antigen.
Ms. Tamara Bock (University of Sydney): transcription factor KLF7 is important for the in vivo expansion and competence of CD19 CAR-T cells in humans with large B cell lymphoma.
Mr Andrew Laskary (QIMR Berghofer): Engineered cardiac patches differentiated from hPSCs in an optimised transplant medium restore cardiac function to baseline following myocardial infarction in murine and porcine models, and show improved function and survival compared to standard media patches.

4. (Left to right) Dr. Selmir Avdic, Production Manager at Westmead T Cell Therapies, and Professor David Gottlieb (Westmead Institute for Medical Research) swapping stories during the tea break.

After a morning tea that buzzed with colleagues connecting over coffee, delegates returned to the auditorium for the Focus on Stem Cells session chaired by A/Profs Ngaire Elwood (BMDI Cord Blood Bank) and Anai Gonzales-Cordero (Children's Medical Research Institute). The first speaker was A/Prof. Elizabeth Ng (Murdoch Children’s Research Institute) whose longstanding mission has been to enable the use of iPSCs for haematopoietic stem cell transplantation. This has been an extraordinarily complex undertaking where Liz and her team went back to first principles – studying haematopoiesis in vivo during embryonic development using fluorescent tags to capture the temporospatial expression of key lineage determinant genes. Their results demonstrated that engrafting HSCs do not arise in the first wave of haematopoiesis, but intra-embryonically from the aorta-gonad-mesonephros at day ~32 of gestation. The work culminated in a 2024 publication that was celebrated by the community as the first systematic method for generating transplantable HSCs from human iPSCs (iHSCs) which can be clinically translated to provide a source of personalised CD34+ iHSCs.

Next, Dr. Megan Finch-Edmondson (Cerebral Palsy Alliance) shared findings on the use of umbilical cord blood (UCB) (which contains MSCs , HSCs and multipotent embryonic-like stem cells) for treating cerebral palsy (CP). Megan began by acknowledging the critical contribution of her co-author who was unable to present, Dr. Madison Paton (a founding ISCT ANZ ESP Subcommittee Co-Chair), and described their findings that long-term persistent inflammation contributes to CP impairment. UCB treatments for CP have been in clinical testing for almost two decades, but recently the field has shifted towards exploring a role for UCB as an anti-inflammatory and immune-modulating modality for promoting endogenous repair pathways following brain injury. Megan, Madison, and the team recently published an individual participant data meta-analysis that sought to extricate clear functional outcomes and dosage parameters from methodologically heterogenous clinical research data. They were able to tease out valuable insights to show that UCB therapy is safe, and results in dose-dependent clinically meaningful improvement in gross motor skills at 6- and 12-months post treatment, particularly in younger patients (<5 years) with milder CP.

5. Associate Professor Anai Gonzalez-Cordero describing the most common cause of vision
loss in the developed world – photoreceptor cell degeneration.

In the final session before lunch, ANZ Vice President Elect A/Prof. Siok Tey (QIMR Berghofer; Royal Brisbane and Women’s Hospital) and Dr. Guy Klamer (Sydney Cord Blood Bank) chaired the session in honour of CGT pioneer and patient advocate, Dr. Geoff Symonds. As a schoolmate of Geoff and Renee’s sons, and a beneficiary of Geoff’s mentorship, Guy opened the Geoff Symonds Oration by reminding the audience that collaboration and mentorship were core values during Geoff’s career. In keeping with that principled approach to scientific discovery, Guy invited the audience to reflect on the question: What are the rational and moral values we share as a society? Dr. Boro Dropulić (Caring Cross; Vector Biomed) then delivered a presentation that honoured his colleague and friend by showcasing what a public-spirited and ethically principled approach to ATMP manufacturing can look like. Using creative business models, Caring Cross (non-profit) and Vector BioMed (public benefit corporation) are driving access to lifesaving CGTs across globe. Boro and his team try to reach poorly serviced areas and lower cost barriers in four main ways:

Technical innovation - Decreasing the intrinsic cost of production by improving materials and processes
Freedom to operate - Using in-house expertise to create highly efficient viral vectors and avoid licensing fees.
Point of care manufacturing - Circumventing the costs of centralised manufacturing (shipping and distribution, personnel, infrastructure, systems) (Tech Transfer Part I).
Training and education - Providing training and education to personnel at point-of-care sites so that products can be manufactured by local staff in local facilities (Tech Transfer Part II).

Using this model, the team at Caring Cross have developed a CAR-T cell pipeline that implements several innovations resulting in a product whose material cost is only USD 20,000 per dose (compared to USD 475,000 for Kymriah, for example). The pipeline has demonstrated good cross-site consistency and is being widely implemented in Brazil in a collaboration with Fiocruz (a foundation from the Brazil Ministry of Health) where Caring Cross’ simplified manufacturing process can generate 250-1,000 CAR-T doses per year in a 65m2 facility on a 5-day per week single working shift. Considering that only 1% of eligible patients are currently receiving CAR-T cell therapy in low- and middle-income countries, this approach could radically increase therapeutic coverage.

Interestingly, Brazil is one of the few countries internationally whose citizens have a constitutional right to healthcare, suggesting that the Fiacruz-Caring Cross collaboration reflects both a moral and rational approach to enacting the value of equitable access to the highest standards of healthcare. In this way, Boro’s presentation provided a glimpse into a possible future where one answer to Guy’s question could be that, if the only ethical application of healthcare is an equitable one, then rationally derived cheap, (relatively) portable, standardised, and simple manufacturing processes are a scientific imperative.

6. Dr. Boro Dropulić describing the $20,000 dose model using Caring Cross’ approach to CAR-T manufacturing.

After lunch, Dr. Sharon Sagnella (Royal Prince Alfred Hospital) and Dr. Hamid Bidkhori (Hudson Institute of Medical Research) introduced a newly established session Engineering the Future: New Strategies in Cell and Gene Therapy Manufacturing (generously supported by Miltenyi Biotec). The first presentation was delivered by Prof. Simon Cool (University of Queensland), who spoke on “Accelerating Australia’s capacity for large-scale manufacturing of life-changing regenerative therapies.” Simon highlighted that, despite the strength of Australia’s research base in CGT and regenerative medicine, the national capacity for large-scale manufacturing lags behind international standards. His presentation outlined opportunities and strategies to strengthen Australia’s position in this global sector. Dr. Mark Allenby (University of Queensland) then expanded on opportunities for improving quality and scalability by integrating computational models into biomanufacturing. He demonstrated how modelling and digital design tools can be applied to create more efficient, robust, and reproducible cell and tissue production systems.

A/Prof. Shayanti Mukherjee (Hudson Institute of Medical Research) then discussed advances in bio-inks for stem cell bioprinting to personalise pelvic floor reconstructive surgery. Her work provided new insights into pelvic muscle physiology and showcased how engineered constructs may accelerate translation of next-generation surgical solutions for urogynaecology. The session continued with A/Prof. Robert Nordon (University of New South Whales) who presented innovations in microfluidic platforms for biologics manufacturing, focusing on strategies to reduce cost while maintaining scalability. Robert and the team have developed a small bioreactor that can capture target cells in channels, house them within small grooves where activation and gene transfer can occur, and then release them for harvesting with a simple switch in the microfluidic flow. Dr. Jingjing (Jane) Li, a talented member of the Nordon Lab, (University of New South Whales) then presented elegant data on her application of these biomimetic microscale bioreactors which attempts to recreate haematological and cardiovascular niches. Using live-cell imaging of hPSC differentiation and single-cell analysis, her team is modelling embryonic haemodynamics to better understand and improve in vitro haematopoiesis.

7. Delegates getting up close and personal with the Nordon Lab’s “GMP in a box” microbioreactor.

Following afternoon tea with exhibitors, the conference reconvened for Expert Insight: Process Development & Manufacturing chaired by A/Prof. Zlatibor Veličković (Cell and Tissue Therapies Western Australia - CTTWA) and Ms Jessica Sue (Sydney Cord Blood Bank). Mr Shane Winzar (Australian Red Cross Lifeblood) lead the presentations by providing an overview of Lifeblood’s product portfolio, including starting material for cell therapies such as PBMCs, and the less popular but equally valuable, faecal microbiota transplant material. He was followed by Dr. Joseph (Yossi) Schwartz (Moffitt Cancer Centre), who highlighted the variability inherent in starting leukapheresis material and the implications this has for downstream manufacturing consistency. Dr. Fran Harding (Therapeutic Goods Administration, TGA) provided practical guidance on regulatory expectations during TGA inspections of laboratories and manufacturing facilities, offering insights into common challenges and compliance requirements.

Dr. Amanda Tan (Kids Research Institute) discussed optimisation strategies for open-system CAR-T manufacturing in the context of an investigator-initiated Phase I clinical trial, underlining the importance of balancing innovation with regulatory readiness. The session concluded with Dr. Daniel Ray (CTTWA), who spoke on quality management systems in cell manufacturing facilities. He emphasised design principles that minimise contamination risk and maintain a fully aseptic environment, highlighting their central role in safe and reproducible clinical translation.

Day One wrapped up with drinks and nibbles during the poster viewing session which featured 57 posters from a variety of categories: Process Development and Manufacturing, MSCs, Exosomes, Regulatory and Quality, Immunotherapy, and iPSCs.

8. (Left to right) Miss Jia Yi Heng (Cytiva), Dr. Huai-En Lu (National Yang Ming Chiao Tung University) and Dr. Waradee
Buddhakosai (E-da Hospital) sharing a smile while discussing work from Waradee and the team
on olfactory ensheathing cells.

Day Two of the conference started off all things regulatory, with moderators A/Prof. Dominic Wall (Cell Therapies) and Dr. Tongted Das (Monash Health), Co-Chair and member of the ISCT ANZ Legal & Regulatory Affairs (LRA) Committee respectively. First, Dr. Giulia Giunti (BioOra) and Dr. Gerry McKeirnan (Increment 4), also members of the ISCT ANZ LRA, gave their perspectives on “Building for Scale: Regulatory Lessons from First-in-human to First-in-commercial”. Gerry presented an overall summary of the push/pull factors in Australia, with a favourable regulatory environment and competent ethics (the push) accompanied by limited facility capacity and high cost of goods sold (the pull). Tips from both Gerry and Giulia included the benefits of planning early, engaging with regulators to align expectations and timelines, planning your manufacturing strategy, and identifying target markets, including what requirements may come with them.

9. Dr. Giulia Giunti sharing her insights on navigating CAR-T regulatory submissions for
clinical trials and beyond in NZ Head of Regulatory Affairs at BioOra.

Following this, the audience heard from regulatory body representatives. Dr. Glenn Smith (TGA) provided a comprehensive summary of when and how the TGA are involved in the development lifecycle of cell and gene therapies. As with Giulia and Gerry, Glenn’s key recommendation for success was engaging with the TGA early. Dr. Geraldine Lester (Office of the Gene Technology Regulator, OGTR) spoke to the role of the OGTR in her talk “An Update on the Regulation of Clinical Trials Involving Genetically Modified Organisms” including how and when their regulation is required, and the remit of the OGTR for CGTs involving genetically modified organisms.

Dr. Karin von Bart (Medsafe) gave the audience an overview of some significant changes happening in NZ, such as the in-development Medical Products Bill which will replace the Medicines Act 1981. Karin outlined NZ’s desire to increase access to therapies for Kiwis and presented the expedited approval pathway for medicines already approved by two overseas regulatory bodies. The theme of early engagement with regulators continued, with Karin reiterating that this is also true for Medsafe approvals. Dr. Tim Strabala (Environmental Protection Agency, EPA) outlined further changes in the regulatory framework in NZ in his talk which discussed the incoming Gene Technology Bill, designed to be standalone legislation that is risk based, allows for efficient approvals and aligns with international requirements. If passed, it will establish a new regulator who oversees the safe use of gene technology, supported by a regulatory team who will process applications and conduct risk assessments.

To round out the regulators, Dr. Joseph (Yossi) Schwartz returned to the podium, this time in his role representing the Foundation Accreditation of Cellular Therapy (FACT). Yossi gave the audience a sneak peak of updates to two sets of standards: the Haematopoietic Standards for Cell Therapies and the Immune Effector Cell Standards, in his talk. In keeping with the times, “transplant” now refers to all cell therapies, and FACT is aiming to harmonise quality and collection sections across the standards. Colleagues who had travelled from across the Indian Ocean were represented by Dr. Ignatius Viljoen from South Africa, who presented “The Regulation of Custom-prepared Autologous Cell-based Products – Is a Paradigm Shift Needed?”, which was an apt continuation of the recent webinar, organised by the ISCT ANZ LRA in collaboration with the ISCT Global Regulatory Task Force.

To conclude the morning session, the regulatory representatives returned to the stage for a panel discussion, giving the audience the opportunity to quiz multiple regulatory bodies simultaneously. Questions from the audience sought further clarification from Medsafe around their expedited pathways, how the OGTR approaches regulation of integrative mRNA (it’s not black and white – reach out to the OGTR is their advice!), regulations around post market surveillance (both the TGA and Medsafe have guidelines available), and the regulator’s positions on heritable human genome editing (ISCT has called for a continued international 10-year moratorium on human heritable gene editing) which is prohibited in both Australia and Aotearoa New Zealand.

10. Dr. Karin von Bart providing an overview of regulatory updates from the New Zealand
Medicines and Medical Devices Safety Authority, Medsafe.

After a morning tea break, speakers returned to the stage for the crowd favourite: Grill the Regulator. Like previous years, they were presented with a scenario: a biotech company has developed an ex-vivo CRISPR-based gene therapy for a rare, life-threatening genetic disorder, whereby the company aim to scale up manufacturing in the local region after initial development/manufacture under a US IND. The panel generously shared their perspectives on this scenario, focusing on appropriate phase classification, whether first-in-human dosing has occurred overseas, and how to manage situations where a manufacturing licence is required locally but it is not needed overseas. Considering that this case was of a rare disease with short life expectancy, both the TGA and Medsafe recognised that platform-based therapies could expedite patient access.

In the last session before lunch, the audience were fortunate to hear from past ISCT President Prof. Bruce Levine (University of Pennsylvania) in the Spotlight on Gene Therapy session (sponsored by Cytiva), chaired by Dr. Janet Macpherson (NSW Health). Bruce provided a comprehensive update of the University of Pennsylvania in development of treatments – analogising advancements in CAR-T cell treatments with those of self-driving cars – and sharing outcomes from UPenn’s phase 1 huCART19-IL18 trial. Demonstrating his characteristic depth and breadth of industry insight, Bruce went onto summarise the field’s understanding of non-relapse mortality after cell therapy (secondary malignancies are also an emerging concern) and provided an in vivo gene editing update: 73 commercial assets disclosed (current to 2024), roughly half of which are viral vector based, and one quarter that leverage LNPs.

Conference Day 2, Thursday 14^th August–Afternoon Sessions

After lunch, A/Prof. Samantha Ginn (President of the Australasian Gene and Cell Therapy Society, AGCTS; Children’s Medical Research Institute) & Dr. Alexandra O'Donohue (Westmead Institute for Medical Research) chaired the ISCT-AGCTS joint session. First up was Prof. Els Henckaerts who presented her work with the Trellis Research Group. The Belgium-based group focuses on improving the efficiency, safety, and accessibility of AAV-based gene therapies. Els began by outlining the key limitations of current AAV vectors, including issues with specificity, potency, toxicity, and immunogenicity, before describing two innovative strategies her team is pursuing. The first involves rational AAV capsid engineering: they developed a novel capsid derived from AAV2 with 14 amino acid substitutions (AAV-TT-hGBA1) that displays enhanced neurotropism and shows promise for targeting GBA1 mutations in Parkinson’s disease. A second strategy involves the addition of targeting moieties to the AAV capsid surface, which achieved up to 10-fold higher CNS transduction and 100-fold improved specificity compared to AAV9, while reducing liver and peripheral organ targeting.

Dr. Grant Logan then shared research from the Children’s Medical Research Institute. Since natural AAV infections generate cross-neutralising antibodies that can block therapeutic AAV delivery, and antibody pre-screening is now a prerequisite for patient eligibility, Grant and the team cloned and characterised monoclonal antibodies (mAbs) from patients treated with Zolgensma (AAV9). Structural mapping revealed that most antibodies target the two-fold axis of symmetry on the capsid, and a small set of amino acid substitutions in AAV9 can markedly reduce recognition in patient sera. These findings, published recently in Molecular Therapy and Nature Communications, represent the largest structural and functional characterisation of human anti-AAV mAbs to date, and provide a blueprint for designing antibody-escape capsids that could expand patient access to systemic AAV gene therapies.

11. Dr. Grant Logan describing their progress towards improving cell therapy for patients
with vector immunity.

Next, the ISCT-ANZ treasurer Dr. Tessa Gargett (Royal Adelaide Hospital) discussed her lab’s work towards improving CAR-T therapy efficacy in aggressive brain tumours by using an AAV-based delivery system capable of producing engineered IL-2 variants directly within the central nervous system. This approach aims to provide localised cytokine support to enhance the persistence and function of CAR-T cells while avoiding the toxicity of systemic IL-2 elevation, and is associated with prolonged survival and tumour clearance in preclinical models. Combining gene therapy with CAR-T cells may enable precise manipulation of the brain tumour microenvironment, allowing sustained T cell activity in a tumour type that has remained mostly resistant to immune therapies.

To close the session, Dr. Jennilee Davidson from Macquarie University described the development of a self-regulating AAV gene therapy for ALS. Jennilee and her colleagues engineered a novel molecular switch that links AAV transgene expression directly to disease state. In human cell culture models, this switch drives expression of a therapeutic transgene only in proportion to nuclear TDP-43 depletion (pathological mechanism in ALS), allowing precise, disease-responsive control. They have paired this system with a cyclin F-based therapy that selectively clears cytoplasmic TDP-43 (which occurs in more than 97% of ALS cases). In preclinical models, the combined switch-therapeutic enabled regulated expression and effective clearance of mis-localised TDP-43, providing proof-of-concept for a precision gene therapy platform that could adapt dynamically to the progression of ALS.

The much-anticipated ESP panel discussion kicked off following afternoon tea, hosted by ANZ ESP Co-Chairs, Dr. Gaurav Sutrave (Westmead Hospital) and Dr. Gemma Moir-Meyer (University of Otago, Christchurch). The session, titled Selling Your Science: From Abstract to Impact, began with a panel discussion featuring Dr. Rebecca Lim (Cell Therapy Manufacturing Centre) A/Prof. Siok Tey and Prof. Dan Weiss. The panellists shared reflections from their own experiences of pitches that were successful (or not so successful!) and critical lessons they learned along the way. They reassured the audience that it was normal for one’s pitching journey to feel messy and uncomfortable but stressed that practice and readiness were key.

Siok encouraged the audience to connect with their stakeholders, develop relationships that allow you to understand their perspectives. Engage with authenticity.
Bec emphasised the power of a clear narrative, recalling a memorable pitch given to her that laid out the problem, summarised what had been tried, identified the gap, and presented a solution with confidence. Uncertain about your pitch’s clarity? Test it out on your friends! Better still, get feedback from colleagues who share similar interests with your stakeholders.
Dan highlighted the importance of being ready with an elevator pitch at any moment, recounting how a chance meeting with a patient’s mother ultimately led to long-term support. Avoid “techno-babble” in favour of accessible storytelling.

This warm and candid discussion provided reassurance that it’s normal for early career pitching to feel awkward (even global President Elect Dan has had his own face-palm moments!), but preparedness (i.e., aligning your message with what matters most to your audience) and practice (i.e., rehearsing your messaging, even if it’s just the first sentence) are key. Ultimately, effective science communication is as much about authenticity and connection as it is about content.

12. (Left to right) Dan, Bec and Siok smiling as audience members try to guess what their
responses were to the question, “The secret to pitching is…?”

The panel discussion ended with a segue into three rapid-fire presentations from local ESPs who demonstrated their science pitching prowess behind the podium.

1) Kah Min Yap (Peter McCallum Cancer Centre; University of Melbourne) described the use of CRISPR/Cas9 knock-in strategies to identify tumour-specific promoters for armouring CAR-T cells.

2) Lakshmy Viswanath (Children’s Medical Research Institute) presented on developing a gene editing approach for congenital adrenal hyperplasia, a life-threatening disorder most commonly caused by CYP21A2 mutations.

3) Michelle Ferguson (Arovella Therapeutics Limited) presented on an allogeneic, off-the-shelf CAR-iNKT therapy targeting CD19.

13. Dr. Michelle Ferguson from Arovella sharing the Melbourne-based biotech company’s
invariant Natural Killer T Cell lead program for allogeneic treatment of haematological
malignancies.

The final day of the conference was a clinical tour de force. Presenters discussed pertinent issues in the clinical application of novel cellular therapies and the strategies being employed to promote equitable patient access in ANZ, but also regionally in Asia and developing countries worldwide.

The Clinical Plenary session, was chaired by A/Prof. Emily Blyth, wearing her clinician hat now, and Dr. David Bishop (University of Sydney). Prof. Cameron Turtle (University of Sydney) kicked thing off by outlining some novel prognostic factors for determining clinical response to CD19 CAR-T cells, with a specific focus on the optimal cytokine conditions required to enhance CAR-T cell anti-tumour activity. He demonstrated the role of IL-15 in enhancing CAR-T cell expansion and persistence, as well as the how reversal of the immunosuppressive tumour microenvironment with PD-1 inhibitors can be harnessed to potentiate the clinical efficacy of CD19 CAR-T cells.

This was followed by two talks from pioneering Australian investigators in the growing field of early phase novel CAR-T cell clinical trials in Australia. Dr. Neevika Manoharan (Sydney Children’s Hospital) presented the results of Levi’s CATCH, a first in human clinical trial of GD-2 specific CAR-T cells to treat paediatric diffuse intrinsic pontine glioma (DIPG). The early results from this study demonstrated safety and some limited efficacy of the GD-2 specific CAR-T cells in an otherwise rapidly fatal disease A/Prof. Siok Tey then provided an update on an Australian first: a phase I clinical trial of a point of care manufactured CD19 specific CAR-T cells. This fresh CAR-T cell product appeared to be safe and has comparable efficacy to the current standard of care CD19 specific CAR-T cell products.

Following on with the theme of home-grown, ANZ region cellular therapies, Dr. Cindy Li (Biosceptre) presented one of the highest scoring abstracts which outlined the pre-clinical development of a modular CAR-T cell system. The design utilises a bispecific antibody containing an antigen binding domain combined with a synthetic, CAR-T cell specific tag. The antigen binding domain provided target specificity, and could be modified to any antigen of interest, thus providing modularity to the system. Meanwhile, the short half-life of the bispecific antibody provides a level of tunability – enabling treatment cessation in the event of toxicity.

The morning session closed with a talk from Dr. Antonio Lee who outlined the history and emergence of Medipost, a company involved in the development of cord blood banking and umbilical cord derived mesenchymal stem cell (UC-MSC) therapeutics in South Korea. Tony presented the development pipeline for the commercialisation of Medipost’s US-MSC products to treat osteoarthritis, including the outcomes from the registration clinical trials and pathway to regulatory approvals in South Korea and Japan.

The second session of the day was Patient Access: From Promise to Treatment and focussed on the critical issue of enhancing equitable patient access for cellular therapeutics, particularly in resource limited locations and for rare indications which lack commercial funding incentives. Dr. Michelle Lorentzos from the Sydney Children’s Hospital spoke on the current landscape of access to emerging CGTs for rare paediatric diseases, with a focus on monogenic metabolic disorders which affect a handful of patients worldwide. Michelle outlined the difficulties in accessing these therapies for patients due to the lack of commercial viability, but also the inability to generate sufficient efficacy data for regulatory approvals. She shared several moving first-hand patient perspectives exhorting clinicians, scientists, regulators and politicians to work together to develop new regulation and access pathways for these diseases.

These clinical perspectives were then juxtaposed with industry insights as Mr Warwick Shaw (Janssen Pharmaceutical Companies of Johnson & Johnson) described how the current framework for regulatory approval and reimbursement can constrain access in ANZ. He outlined plans for modifying the Health Technology Assessment legislation to allow for enhanced access to treatments for patients with rare diseases and proposed potential funding models to reduce financial barriers to access. Ms Sharon Winton, the CEO of Lymphoma Australia, then described the need for ongoing advocacy to ensure that new treatments and clinical trials are available for all patients across ANZ, and to reduce the impact of geography and socioeconomic status. She provided heartbreaking examples of patients who narrowly missed out on access to therapies that had received favourable Medical Services Advisory Committee recommendations but were unavailable due to funding negotiation delays. The session concluded with a round table discussion led by the three aforementioned speakers who fielded questions from the moderators (Dr. Joanna Youngson from Alfred Health and Mr Dominic Fernandez from Epworth) and the audience. The wide-ranging discussion highlighted how the differences in regulatory frameworks, legislation, and resources influence the availability and access to CGTs around the world.

14. (Left to right) Associate Professor Emily Blyth, Dr. Joanna Youngson, Ms Sharon Winton,
Dr. Michelle Lorentzos, Mr Warwick Shaw and Mr Dominic Fernandez standing together after
an expansive discussion on improving patient access.

Finally, A/Prof. Siok Tey and Prof. Dan Weiss chaired Cell Therapy from Asia to the World, which was a fantastic session to conclude the meeting, and outlined some of the amazing progress being made in the clinical applications of CGT in Asia. Dr. Pawan Gupta from Stempeutics Research in Bengaluru provided the clinical outcomes from two different Indian CD19 CAR-T cell therapy products, ImmunoACT and QARTEMI. These products both demonstrate similar efficacy and safety to the TGA approved CD19 specific CAR-T cells products (tisagenlecleucel and axicabtagene ciloleucel), however, they can be manufactured at a fraction of the cost and onsite at primary healthcare facilities, the cost making them eminently more accessible in the Indian socioeconomic context. He also provided the outcomes from the clinical trials of two other MSC products developed by Stempeutics, including Regenacip for critical lower limb ischaemia and Stempeucel for osteoarthritis, highlighting the expanding footprint of CGT in the South Asia region. Pawan was followed by Dr. Sarah Ho from the National University of Singapore who outlined the CGT landscape in Singapore with a focus on gene modified MSCs and how these therapeutics have navigated the pathway from in vitro evaluation to clinical trials. The session was rounded out by the highest scoring abstract from the Asia region which was submitted by Dr. Harry Murti from Kalbe Pharma in Indonesia who provided a thorough characterisation of development of a GMP compliant process to reprogram umbilical cord derived MSCs into an iPSC bank for use in various applications.

15. Dr. Pawan Gupta (ISCT Asia Regional Vice President) describing the similar safety and
efficacy profiles of Indian CD19 CAR-T cell products to TGA-approved equivalents.

After an informative and moving final day, A/Prof. Emily Blyth and A/Prof. Siok Tey closed the meeting with the presentation of awards, with the Best Abstract Presentation to Dr. Andrew Laskary, the Best Early Stage Professional Abstract to Dr. Jennilee Davidson and the Best Technologist Abstract awarded to Ms Natasha Barry.

16. Best Abstract Presentation - Mr Andrew Laskary.

17. Best Technologist Abstract - Ms. Natasha Barry.

18. Dr. Jenilee Davidson (right hand side) receiving her award Best Early Stage Professional
Abstract from Siok (left) and Emily (middle).

With attendance that exceeded projections and spanned beyond the region’s core bearings, the ISCT ANZ annual meeting demonstrated the field’s rapid expansion in the Global South. Given the resource intensive nature of CGT manufacturing, ensuring equitable and sustainable access to these treatments will require close collaboration across all sectors. There is much that can be learned from international efforts and adapted for an ANZ context (e.g., Dr. Frank Li’s article on Hospital Clínic de Barcelona), particularly de-centralised manufacturing processes, de-risked tech transfers and tight partnership with regulators. The community was as collegiate and collaborative as ever, and the organising committee look forward to seeing everyone together again in Melbourne, June 2026!

19. ISCT ANZ Regional Meeting Organising Committee.
Top left: Tessa Gargett; Top right: Siow Chan.
Back row (left to right): Leon Brownrigg, Dominic Fernandez, Zlatibor Veličković, Selmir Avdic,
Robert Nordon, Ali Shokoohmand;
Middle row (left to right): Alicia Didsbury, Maddie Olley, Liv Purnell, Emily Blyth, Francisco Chung,
Janet Macpherson;
Front row (left to right): Gemma Moir-Meyer, Ngaire Elwood, Siok Tey, Giulia Giunti,
Joanna Youngson, Cheryl Hutchins.

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