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Safety concerns spur FDA to again halt testing of Regenxbio gene therapy

Published

24 August 2026

Topic

opportunities

Sectors

Biotech

Geography

United States

Source

Read at biopharmadive.com

Verified

Fusion42 · 25 August 2026 · Fusion42 review

The FDA has placed a second clinical hold this year on Regenxbio's gene therapy for Hunter syndrome after detecting safety concerns involving spinal masses in five patients.

This Wire brief sits within Fusion42's coverage of Biotech.

◆ The Wire takeaway

Gene therapy founders focused on rare diseases must treat FDA holds as urgent access blockers and adjust trial safety protocols immediately. This pause signals tightening FDA scrutiny that could delay market entry and increase development costs.

Coverage

1 source · 24 Aug 2026

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Topics

Biotechgene-therapyclinical-holdFDAsafety-concernsregenxbio