Cell and gene therapies represent the “next evolutionary step” in bioprocessing incorporating disruptive technologies on a well-proven manufacturing base, agree panelists at BPI Europe.
“Is is cell and gene therapy really the shiny, bright object on the field that breaks all the rules and disrupts everything? Or is it just an iteration of the same principles that we had before, with slightly different practices?” That was the question Nadine Ritter, president and analytical advisor at Global Biotech Experts posed to a panel of experts in Vienna, Austria this week.
The panel was the plenary at BPI Europe — the first to be held in person since 2019 — and, as expected, the answer was not clear cut.
“There are elements which are really disruptive and different than the things we have done so far,” began Dirk Böhm, head of CMC & RA within the Cell and Gene Therapy unit at Bayer Pharmaceuticals. He stressed that while many processes can be taken from the well-proven biologics playbook, areas such as understanding the process and the analytics surrounding that need a lot of work.
He noted the difference between cell therapies where the “application of living cells is a much more complex endeavour than what we have currently in the biologics environment” and gene therapies, where the “entering and modifying the genome, fixing gene defects, and having the right controls and the right means to make sure this is all working well, is a very different thing” — a key difference picked up by others on the panel.
Amandine Breton, associate director of Cell Process MSAT at Orchard Therapeutics, said there is potentially a more disruptive approach in cell therapies than gene therapies, which can benefit more from traditional approaches developed in the small molecule, vaccine and biologic fields.
“When you start considering the cells, you’re really into the domain where it’s a life product that is moving and changing, and you need to adapt to that.” She noted the tailor-made nature of autologous cell therapies and how a batch is made for each patient. “You have that variability to encompass,” she said, reiterating the need for “stronger analytical tools to help you embed that variability.”
Jenny Ann Prange, CSO at MUVON Therapeutics, which is developing an autologous cell therapy to treat stress urinary incontinence, agreed with her co-panelists but added such advanced therapies represent “the next evolutionary step” when it comes to processing.
“There are a lot of methods that are already established. They have to be adapted, obviously, and there has to be a lot of work to be done there. But in general, I think the base is already there.” Again, she differentiated between therapy types, specifically autologous and allogeneic, saying for personalised medicine “there is no solution that fits every question that there is, so it has to be adapted in the end.”
Defining disruptive technologies as as a combination of different scientific, physical, mathematical, and biological horizons,
Yasser El-Sherbini, a CMC and regulatory consultant from Biopharma Excellence said cell and gene processes are based on those that have come before but disruptive technologies are being slowly introduced to overcome hurdles. He said new technologies such as in silico process development, which is being introduced by some developers, are beginning to transform the industry. “I would say it’s iterative, but it’s becoming over time, more disruptive,” he concluded.
However, while moderator Ritter welcomed the necessity of disruptive technologies, she stressed “we should not confuse disruptive technology with the rules that are associated with safe, quality, efficacious medications.”
She continued, adding that “disruptive technology for producing these materials and for the mechanism of action is fantastic… But we have to produce them and control them and monitor them in alignment with the rules that have been established to protect public health and safety, because the worst thing that can happen is that we find this fantastic approach and we harm people inadvertently and It kills the technology before it can go forward.”