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# FDA Orphan Status for Vision Loss Gene Therapy
- URL: https://www.fdaweb.com/fda-orphan-status-for-vision-loss-gene-therapy/
- Published: 2017-08-04T12:00:00.000Z
- Updated: 2026-09-14T22:39:07.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5139310

FDA has granted Applied Genetic Technologies an orphan drug designation for its unidentified gene therapy product candidate for treating X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene. XLRP is an inherited condition that causes progressive vision loss, beginning with night blindness in young boys followed by progressive constriction of the field of vision, according to the company. Preclinical data indicate that treatment with the gene therapy product slowed the loss of visual function in a canine model of XLRP due to mutations in the RPGR gene, it says.