Regulatory ShiftDeveloping
The FDA May Be Opening a More Flexible Path for Rare-Disease Gene Therapies
A new FDA draft guidance and a series of recent reversals suggest the biggest bottleneck in gene therapy may no longer be the science alone. It may be the approval process itself.
Why now
Draft guidance and recent reversals suggest the rare-disease rulebook is changing in real time.
- Overclaim risk
- medium-high
- Primary source
- Regulatory
- Published
- Jun 24, 2026
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