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.
Comments
Viral vectors such as AAV continue to struggle.