FDA again halts Regenxbio gene therapy trial over safety concerns

The U.S. Food and Drug Administration (FDA) has again halted clinical trials of Regenxbio’s gene therapy for a rare childhood brain disorder. The reason was small lesions detected in the spinal area of five patients.

Lesions in five patients

As STAT News reports, all five patients have mucopolysaccharidosis type II, also known as Hunter syndrome. According to Regenxbio, the patients had no symptoms associated with these lesions.

The company announced the new trial halt on Monday. The FDA suspended the study due to concerns about the therapy’s safety.

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Previous case of a brain tumor

The announcement came seven months after Regenxbio said that a boy who received a very similar therapy developed by the company to treat mucopolysaccharidosis type I had developed a brain tumor.

According to the publication, this was the first case in which a tumor was definitively linked to adeno-associated virus (AAV)-based gene therapy. This approach is the most common form of gene therapy and has been used over the past 20 years.