GovernmentU.S. health regulator
- Source type
- Government
- Access type
- Official
- Publisher
- U.S. Food and Drug Administration
- Date
- 2023-12-08
- Added
- 2026-06-27
Trust profile
94
U.S. health regulator
- Publisher type
- Regulator
- Bias profile
- Low
Best used for
- Approvals
- Labels
- Safety communications
Weak for
- Comparative effectiveness
- Consumer experience
Used in Viral Vitalism
Regulatory statusbrief1 reference
McKenzie Is Preparing to Become CHLA's First Casgevy Patient
Roles: Regulatory status
Show section-level references
Regulatory statusbrief1 reference
A 23-Year-Old in Louisiana Was Functionally Cured of Sickle Cell Disease
Roles: Regulatory status
Claims supported
No reusable evidence claims are currently attached to this source.
Related studies
No structured study record is currently attached to this source.
Related sources
GovernmentU.S. health regulator
FDA: Draft Guidance to Help Accelerate Cell and Gene Therapies
Canonicalized from rapid brief fda-rare-disease-gene-therapy-flexibility.
- Trust score
- 94
- Publisher
- U.S. Food and Drug Administration
- Access
- Official
- Usage
- 1 connection
GovernmentU.S. health regulator
FDA: Leveraging Prior Knowledge in Gene Therapy Products Incorporating Genome Editing
Canonicalized from rapid brief fda-rare-disease-gene-therapy-flexibility.
- Trust score
- 94
- Publisher
- U.S. Food and Drug Administration
- Access
- Official
- Usage
- 1 connection
ArticleGeneral published source
Fox 8 / WVUE: New Orleans man becomes first in Louisiana functionally cured of sickle cell disease
Canonicalized from rapid brief daniel-cressy-sickle-cell-gene-therapy-louisiana.
- Trust score
- 60
- Publisher
- Fox 8 / WVUE
- Access
- Publisher
- Usage
- 1 connection
ArticleGeneral published source
The Guardian: Louisiana man functionally cured of sickle cell disease
Canonicalized from rapid brief daniel-cressy-sickle-cell-gene-therapy-louisiana.
- Trust score
- 60
- Publisher
- The Guardian
- Access
- Publisher
- Usage
- 1 connection
ArticleGeneral published source
Reuters: UniQure to seek approval for Huntington therapy after FDA U-turn
Canonicalized from rapid brief fda-rare-disease-gene-therapy-flexibility.
- Trust score
- 60
- Publisher
- Reuters
- Access
- Publisher
- Usage
- 1 connection
ArticleGeneral published source
Regenxbio: Alignment with FDA on path forward for Navsunli BLA resubmission
Canonicalized from rapid brief regenxbio-navsunli-rgx-121-fda-accelerated-approval-path.
- Trust score
- 60
- Publisher
- Regenxbio
- Access
- Publisher
- Usage
- 1 connection
Topic tags
medicineregenerative-medicinesickle-cell-diseasegene-therapycasgevycrispr