FDA Gene Therapy Approvals (1998-2025): Current Status, Regulatory Evolution, and Future Directions.
Systematic review documents over two decades of FDA gene therapy approvals, providing a roadmap of how rare genetic diseases now have treatment options.
This systematic review maps the complete landscape of FDA-approved gene therapies over 27 years, capturing the regulatory evolution that enabled approval of treatments for rare genetic diseases. It provides a high-level reference for understanding the current state of gene therapy commercialization and outstanding translational challenges.
What the study was
- Study design
- review
- Population
- FDA-approved gene therapy programs (1998-2025), predominantly rare disease
- Category
- Drug Development
- Maturity
- Validated
- Journal
- J Clin Pharmacol
Why it surfaced
Standard: useful landscape overview for rare disease gene therapy tracking; covers a core Pulse watchlist area; review design limits direct clinical impact; published in a credible pharmacology journal.
A plain-language summary of published research — not medical advice. Talk to a clinician about your own care.