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ISCT ANZ 2024 Gene Therapy Manufacturing Workshop: A Review

  

Leon Brownrigg, PhD
ISCT ANZ Regional Secretary 2023-2026
Senior Medical Scientist
Cell and Tissue Therapies WA (CTTWA)
Perth, Australia

A magnificent setting and the view behind the day’s presentations: Mountains over Lake Wakatipu, Queenstown, Aotearoa New Zealand, 06 AUG 2024. (Photo: L Brownrigg)

It was a cool, crisp and sunny winter’s morning that greeted forty-five participants at the dawn of the first ever in-person cell and gene therapy manufacturing workshop held in the Australia and New Zealand (ANZ) region. The venue was the Heritage Hotel, nestled on a vantage point overlooking serene Lake Wakatipu and the majestic snow-capped peaks that envelop Queenstown, Aotearoa New Zealand. The course spearheaded ongoing ISCT workforce development activities in the region. It promised a full day of seminars presented by experts from accredited commercial and not-for-profit manufacturing facilities, and Contract Research Organisations from all over ANZ. This was not a purely academic exercise; this was training backed by real-world experience.

The event was convened and facilitated by Zlatibor Veličković (Cell and Tissue Therapies WA) and included presentations from Gerry McKiernan (Increment4), Julie Jones (BioValeo), Rajiv Khanna (Q-Gen Cell Therapeutics, QIMR), Melanie Domingues (Cell Therapies Pty Ltd), and Kelly Hunter (Orthocell). True to the nature of the region’s inhabitants, the sessions were lively, interactive, relaxed and egalitarian, with insightful questions and discussions generated by attendees and presenters alike.

Event content covered the complete range of manufacturing experience from pre-clinical to market post-Phase II. Each unit of the curriculum had well-defined and developed learning objectives. The day opened with presentations on the regulatory frameworks encountered in each of the major regional jurisdictions, where Gerry McKiernan gave the Australian story and Julie Jones provided the local NZ perspective. 

Rajiv Khanna then gave a series of presentations on the principles of GLP, preclinical considerations for investigative cell therapies, and Phase I clinical trial design.

Principles of GMP for Phase II clinical trials were presented by Zlatibor Veličković, who also went on to cover cleanroom operations, aseptic manufacture, cell sources and materials management in greater depth.

Modules on cell processing and manufacture including genetic modification were delivered by Melanie Domingues. Melanie then guided learners through units on analytical methods, quality control, documentation, batch records, logistics and supply chain management.

Gerry McKiernan returned to give an overview of risk management and quality systems, including a memorable analysis of risk and one’s probability of survival when swimming with saltwater crocodiles of various sizes to really drive a point about the value of information and expertise. The Safety Officer in me still advises readers that risk elimination comes before risk mitigation, so please don’t swim with crocodiles as they are very good at what they do.

Julie Jones presented the modules on good clinical practice (GCP), clinical operations and study management, including sections on data integrity, auditing, patient rights, safety and wellbeing. Then the day’s curriculum was brought to a close by Kelly Hunter who delivered the module on regulatory compliance beyond Phase II, including regulatory submissions, pathways, approvals and post-market commitments.

There was a great deal of material covered in this event. Every module could naturally have easily filled a half-day of content by themselves, and the coordinators did extremely well to keep the modules concise and the learning objectives on point.

The intention is that this workshop will be held annually in conjunction with the ISCT ANZ regional meetings, and that all future iterations of this workshop will be fully accredited. The next workshop will be held at ISCT ANZ 2025 Sydney. Do all that you can to ensure that you or your mentees are there for it.


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