Developers of gene therapies should use tactics de-risked by others, such as using better-known capsids, to move faster through to market. That’s according to Adrien Lemoine, co-founder and CEO of Bloomsbury Genetic Therapies.
Lemoine will be speaking at LSX Congress in May on a panel about CGT commercialization, including developing a seamless manufacturing plan.
According to Lemoine, only a handful of gene therapies have been approved, despite a great deal of time and effort.
“The main reason it’s been taking so long to get AAVs [adeno-associated virus vectors] for gene therapies approved has been primarily safety and manufacturability,” Lemoine explains.
“And a common denominator of safety and manufacturability issues with gene therapy is the capsid.”
Choosing a capsid that is part of an approved product or is already a long way into development in another company, he explains, can help move the product faster into the development, manufacturing, and approval process.
This is especially the case with orphan diseases where it’s not economical to spend ten years developing an AAV, he says.
“If you start with a known, tested capsid that allows you to do much slimmer, more compact development because regulators will have seen those capsids many times, and you don’t need to redo all the work,” he says.
Well-known capsids will have already de-risked manufacturability because they’ve already been made successfully at the right titers/volumes, especially when the AAVs are manufactured at a CDMO who has already got gene therapies approved for the market.
“Some programs have started with [viral] vectors made in an academic center, and they were very far from commercial capabilities,” he says.
“If that happens, you will need to change your source of supply from your study to commercial manufacturing, and—if the difference is too big—regulators will challenge whether the vectors are comparable.”
He argues that making your vector in a commercial facility during early-phase development reduces the chance that your clinical material and commercial vector are not comparable.
“That’s our approach,” he says. “And we are working with a CDMO who has a commercial footprint and will be making our first GMP batch.”
