Creating a genetic medicine manufacturing ecosystem: part 5

John Lennon¹ laid the foundations for a global movement for peace when he wrote ‘Imagine’. The lyrics are well suited to the struggle to gain equity for medicines around the planet, in a sense democratising medicine.

Australia can raise its head above our historical antipodean cringe and realise we are no longer subservient to our colonial heritage, nor should we feel we are subordinate. We have a material opportunity to affirm our destiny and be a global leader.

We all can cite our Nobel Prize-winning rock stars, but we need to give airtime to the legion of scientists surviving their legacy in Australia, despite the lack of resources and national support. COVID-19 had drawn public consciousness, never before experienced, to the importance of scientists. It highlighted their talent and, importantly, their role in combating false claims and pseudoscience. This is my fifth article in a series that started in April 2021², where we were imagining an Australian genetic medicine manufacturing ecosystem.

In a recent article in Lab+Life Scientist, ‘COVID-19, vaccines and pandemic preparedness: lessons learnt’³, three prominent vaccinologists look back at the challenges they faced early in the pandemic. There is a common thread that runs through this article: the importance of being ready. Professor Sarah Gilbert, who initiated and led the production and development of the Oxford–AstraZeneca vaccine (Vaxezevria), had started to ask the question in early 2019 “how would we move from the idea through to actually having a vaccine in clinical trials in a really short space of time?” By April 2020 they had already completed a dozen clinical trials of viral vector vaccines prior to pivoting to COVID trials. Clearly having all the systems in place, the expertise, the teams and knowledge of the regulatory and ethic environments fast-tracked their vaccine. They moved from speed being the issue to, interestingly, funding!

In Australia, The University of Queensland’s Professor Paul Young had been developing molecular clamp technology for some eight years; they were funded by the Coalition for Epidemic Preparedness Innovations (CEPI) to develop the platform to target MERS (Middle East respiratory syndrome). A year into it, CEPI asked them to focus on COVID. Professor Terry Nolan stated, “That so-called pandemic preparedness, and having those sort of platforms available, is really the most important insurance that we can do in the research sector to be prepared for whatever might unexpectedly turn up in the future.”

Looking at what was broken during the pandemic brings into sharp focus our reliance on the rest of the world to survive; the supply chains we had become so accustomed to fell over. While there was global scientific collaboration, it was difficult to get the required consumables for many critical pieces of equipment, as scientists clamoured to secure a sufficient supply for their research. It is clear that, for this ‘ecosystem’ to develop, we need not be too prescriptive or strategic; we need to support a myriad of organisations to carve a niche in capacity for this country. What is happening presently is, the very scientists at the core of the movement I mentioned in my first article in this series have had a hand in much of the infrastructure being commissioned. They are continuing to go their own paths, ensuring a broad spectrum of research and development of those critical skills of GMP production. Importantly, it is not just academia, as there is an emergence of CMOs (contract manufacturing organisations) and CDMOs (contract development and manufacturing organisations) emerging in Australia.

There is something truly special brewing in Melbourne, a hugely powerful collaboration of critical institutes to build what arguably will be the flagship for this country — the Australian Institute for Infectious Diseases. The collaborations are not simply those organisations that will be housed in the bricks and mortar, but the national and international respect and collegiate attitude these people convey. There is a concept for a full GMP facility to be included in the construction, and this is true leadership — learning from the past, understanding the future needs. They have grasped the point: we need the experience of walking the path and practising the steps before we must run at warp speed.

It is well and good to have the infrastructure; it is equally critical to have the basics in place. The delivery formulation of medicine is fundamental. It is apparent that even clever people make mistakes; they can have great technologies but still fail. During a meeting with a GMP scientist at a prominent US pharmaceutical company recently, it was stated “This is where formulations come to die.” He was extolling his frustration with the protocols that are developed in a lab but fail to translate to scaling. In mathematics it is obvious that a small calculation may have acceptable errors, so scale it up and you multiply the errors.

Why do these technologies have issues? Insight can be gained in the recent article ‘A better way to create nanoparticles for in vivo treatments’⁴, where it is evident that the core design of the technology is as crucial when starting the journey as it is when the medication is produced. Comprehending maintaining identical conditions in the lab to GMP suite makes infinite sense, thus eliminating the ‘death’ of formulations. Reviewing the offerings of all available technologies, Advanced Cross Flow (AXF)⁵ is arguably the winning concept.

We must not forget that, when investing in a platform technology, sustainability is paramount. The cost of consumables to operate is one facet; can we get these during pandemic conditions? Selecting technologies that are not reliant on others will be the key.

Evidenced by the academic focus on the production of genetic medicines, they are taking the lead, imagining! Years ago, I thought I was a dreamer — I was not the only one. It is only when the ecosystem is healthy that we can truly democratise medicine.

_{1. ‘Imagine’ John Lennon https://youtu.be/YkgkThdzX-8?si=RSLZsMUhbL79T6k4}

_{2. ‘Creating a Genetic Medicine Manufacturing Ecosystem’ Peter Davis https://issuu.com/westwick-farrowmedia/docs/lab_and_life_scientist_apr_may_2021/6}

_{3. ‘COVID-19, vaccines and pandemic preparedness: lessons learnt’ Lauren Davis https://issuu.com/westwick-farrowmedia/docs/lab_and_life_scientist_dec_2023_jan_2024/6}

_{4. ‘A better way to create nanoparticles for in vivo treatments’ Peter Davis https://www.atascientific.com.au/a-better-way-to-create-nanoparticles/}

_{5. Advanced Cross Flow (AXF) https://microporetech.com/our-services/crossflow-technology}

Image credit: iStock.com/Smederevac