Manufacturing
Standardising quality and pre-clinical testing requirements could reduce the cost of developing CGT products.
speaks volumes about the industry’s desire for better standards and regulatory clarity.” Even better, successful applications to the FDA for human testing will be made publicly available, making it easier for other manufacturers to follow in their wake. There are clear practical advantages to this approach – especially to the most vulnerable patients. It could mean, after all, that individuals with the rarest genetic disorders might be able to receive treatment before the traditional three-stage development cycle is completed. That’d surely be a godsend to sufferers of numerous diseases, notably Huntingtons, whose genetic cause was first pinpointed in 1993, but where medical progress has lagged. More than that, Lee explains that these ideas could be applied right across the CGT sector. “By developing a platform that is productive, reliable, robust, and well-understood,” he says, “one can imagine using the same platform over and over to produce medicines to treat different genetic diseases by only changing out the ‘gene of interest’, the result being greater regulatory confidence in the approach.”
“I think we are right on the cusp of CGT having a major impact for patients, on par with where antibody-based therapies were 30 years ago.”
Courtney Silverthorn, AVP for research partnerships at the FNIH
Manufacturing isn’t the only area that could be shaped by the BGTC. Upstream of production comes R&D, with Silverthorn claiming that collaborative research on the basic biology of adeno-associated viruses (AAVs) could inform how vectors from these viruses are made in future. That’s shadowed, she continues, by bolstering the efficiency of AAVs, for example lowering their viral loads while still retaining their effectiveness. Given all this, it’s no wonder that Lee is optimistic about the future of the BGTC. “There will always be organisations that will protect their own
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innovations,” he concedes. “But convergence on manufacturing platforms can ‘lift all boats’ – as was experienced with the biopharmaceutical industry’s growth after convergence on an antibody manufacturing platform.”
Other sectors, other vectors That last point is worth dwelling on. For if it’s clear that the sector has already witnessed success around the manufacture of antibodies – in 2020, Eli Lilly and Amgen announced a manufacturing partnership, drastically increasing the global supply of Covid-19 therapies – there’s no reason the same principles can’t be applied even more widely. As Silverthorn stresses, the collaborative Accelerating Medicines Partnership model epitomised by the BGTC has worked well in other areas of medicine, including Parkinson’s, Alzheimer’s and schizophrenia, with more projects currently in development. Nor is this especially surprising; with personalised medicine set to become a $922bn industry by 2030, siloed manufacturers focused on large-scale production may struggle to cope. Not, of course, that old-school manufacturing seems destined to vanish altogether. In Silverthorn’s telling, the point of public-private partnerships like the BGTC is to “supplant, rather than replace” the sector’s current production format – a claim supported by the facts. In the United States, a subsidiary of Roche recently announced plans for a $575m gene therapy innovation centre in Philadelphia. On the West Coast, German giant Bayer has earmarked $200m for a bespoke cell therapy plant in Berkeley. In whatever form it comes, though, both Silverthorn and Lee agree that the future for CGTs looks very bright indeed. As Silverthorn puts it: “I think we are right on the cusp of CGT having a major impact for patients, on par with where antibody-based therapies were 30 years ago.” Considering the field itself is little more 30 years old, that’s a remarkable thing to hear. ●
World Pharmaceutical Frontiers /
www.worldpharmaceuticals.net
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