AGTC Files IND For Treatment Of X-Linked Retinitis Pigmentosa - Quick Facts

(RTTNews) - Applied Genetic Technologies Corp. (AGTC) said it has filed an Investigational New Drug application or IND with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company's gene therapy product candidate for the treatment of 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. Affected men progress to legal blindness by their early forties.

According to the company, preclinical data indicated that treatment with an investigational gene therapy product slowed the loss of visual function in canines with XLRP caused by mutations in the RPGR gene.

XLRP and X-linked retinoschisis (XLRS) are the two lead development programs within AGTC's collaboration and license agreement with Biogen, Inc. (BIIB), to develop gene-based therapies for multiple ophthalmic diseases.

AGTC said it plans to initiate a clinical study evaluating the safety and efficacy of its proprietary gene therapy for XLRP caused by mutations in the RPGR gene in the U.S. in the coming months.