Over the three days of Advanced Therapies Week (ATW) 2023 in Miami, Florida, US, from 18 to 20 January, DDW’s Megan Thomas heard from attendees about what they were most looking forward to learning from the event, as well as why they think the potential of cell and gene therapies outweighs the obstacles that must be overcome to achieve it.
Matt Lakelin, co-founder of TrakCel, shared that although ATW took place last year, 2023 sees the first fully subscribed ATW since the coronavirus pandemic. Before the event, he said: “I am most looking forward to catching up with some old friends in the cell and gene therapy industry face-to-face that I have not seen for a long time. One of the many great aspects about working in the cell and gene therapy industry is the spirit of collaboration and I will be using 2023’s event as a catalyst for collaborative programmes.”
Lakelin thinks the profound clinical effects of cell and gene therapies will always provide a driving force for the industry to overcome the obstacles which complicate management and administration of these products.
Luke Thorstenson, Director of Business Development at Orgenesis, thinks that the event does a great job of bringing together the companies developing processing and manufacturing platforms with the therapeutic developing entities who need these technologies to enable true scale of cell and gene therapies. He said: “In addition to the great showcase of exciting new technology, the programming of the event has identified trending platform opportunities such as the panel on decentralising advanced therapies..”
Dave Yoder is Senior Manager, Product Strategy at Aldevron. Yoder said: “Aldevron is excited by the opportunity to interact with C> community to learn about new technologies and initiatives so we can continue to help enable new, lifesaving therapies. As technologies evolve, the need to manufacture the components of new drugs under a GMP quality system remains and we are ready to support.”
Yoder hopes that attendees will recognise that the field of C> is continuing to rapidly evolve with new technologies to overcome a variety of obstacles and that new collaborations will develop to bring more therapies to the clinical space. He said: “The potential to bring lifesaving therapies that go beyond treating a condition to enabling a cure certainly outweighs any obstacles. The medical and scientific communities, in collaboration with GMP manufacturing partners, must continue to find ways to leverage cell and gene therapies to develop new treatments for those in need.”
Naiara Tejados, Phd, Head Of Technology Development at VIVEBiotech, shared that she was most looking forward to learning all the steps that are being taken towards the commercial-scale manufacturing of advanced therapies. She said: “It is exciting to see how an increasing number of them are being administered to patients. For me, from the manufacturing point of view, the most relevant issues to be addressed are the ones linked to the establishment of increasingly more controllable, optimised and cost-effective manufacturing processes. This would result in making these therapies more accessible to a greater number of patients suffering from even more prevalent diseases.”
Tejados says that some diseases have no treatment options and that cell and gene therapies could definitely cure many of them. She said: “So, they are the clear example that any obstacles can be outweighed by these advanced therapies. Additionally, in the case of various chronic diseases, there are only palliative treatments and patients do not have a good quality of life. For all these diseases requiring expensive lifelong treatment, cell and gene therapies represent a potential curative solution that may, in many cases, offer substantial savings.”
Professor Ivan Wall, Head of the Centre for Advanced Therapies Manufacturing Training at the University of Birmingham, is most looking forward to seeing what advances in digital and data technology have happened since the last meeting. He said: “We still have a long way to go in creating and deploying systems that adequately capture, interrogate and then use data to make better products. There is a real opportunity for the sector to come together and identify those challenges and then respond to them in a coordinated fashion and ATW is the place to kickstart the discussions.”
David Wellis is the CEO of Excellos, thinks the potential of cell and gene therapies outweigh the obstacles because: “First, we know it works; and second, because, as these therapies have evolved through years of research and innovation, the obstacles have become better understood, moving from ‘unknown unknowns’ to ‘known unknowns.; This allows the industry to more productively address these obstacles throughout the journey from basic research to commercialisation and treatment delivery.” He hopes that attendees will leave ATW 2023 understanding that the issues that stand in their way are being addressed across the board, from the quality and effectiveness of the raw material to ensuring that these attributes are unaffected by the manufacturing process itself.
Dr Edwin Stone, CEO of Cellular Origins, thinks that cell and gene therapies harness the most sophisticated tool that we have in the body to combat disease. He said: “It is this that I believe enables it to tackle diseases that traditional small molecule and biological approaches cannot. It is a new age of medicine. We have overcome so many biological hurdles and continue to do so. When we look at the remaining challenges, perhaps key among these is getting them to patients at acceptable prices, for which we have a whole range of tools that we are only just starting to deploy.”
Stone believes that shows like ATW demonstrate how much more we can do. He said: “The biology will continue to move forward to support this, whether by reducing manufacturing time or by allogeneic approaches, the payer environment continues to change to find more appropriate ways to reimburse novel therapies and manufacturing technology is eliminating labour. Perhaps the main thing that encourages me is that the industry recognises we have to find a way to overcome the challenges, ensuring a motivated eco-system intent on finding the answers.”
Sagi Nahum, Senior Director of Analytical Sciences and CMC, CGT, Orgenesis, believes that cell and gene therapies have the potential to revolutionise medicine by providing personalised and highly effective treatments for a wide range of diseases targeting specific cells and genes, allowing for more precise treatment. Despite this potential, he notes that they are still not available or affordable for most patients in most cases.
He said: “CAR-T/CGT centres are not currently available in every hospital and CGT product costs are high, caused by high production (materials, facilities and labour) and logistic costs (shipment and storage). For autologous products, decentralised production has strong potential to be one of the ways to overcome this obstacle. Local cell production in small GMP facilities shows promise to reduce manufacturing costs and minimise shipment and logistics costs. In addition, innovations in automation and robotics should reduce labour and increase product manufacturing efficiency and safety. Finally, new AI tools are going to be available soon to improve the cell manufacturing process and control to achieve higher yields with enhanced cell therapeutic activity.”
