> The announcement comes seven months after Regenxbio announced that one boy who received a very similar therapy the company developed for MPS type I developed a brain tumor. It was the first tumor to be conclusively linked to AAV gene therapy, the form of gene therapy most commonly used over the past 20 years.
> The FDA halted both the MPS I and MPS II trials at the time. And it rejected an application for the MPS II therapy in February.
Viral vectors such as AAV continue to struggle.
> The announcement comes seven months after Regenxbio announced that one boy who received a very similar therapy the company developed for MPS type I developed a brain tumor. It was the first tumor to be conclusively linked to AAV gene therapy, the form of gene therapy most commonly used over the past 20 years.
> The FDA halted both the MPS I and MPS II trials at the time. And it rejected an application for the MPS II therapy in February.