AGTC Receives US FDA Orphan Drug Designation for Gene Therapy to Treat X-Linked Retinitis Pigmentosa

August 11, 2017: By Jon Swedien

AGTCApplied Genetic Technologies Corporation announced Aug. 10 that it has filed an investigational new drug application (IND) with the US FDA to conduct a Phase I/II clinical trial for the company’s gene therapy candidate for X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

AGTC said it plans to start the study in the US in the coming months.

AGTC is currently scheduling patients for enrollment in clinical trials for X-linked retinoschisis (XLRS), as well as for achromatopsia caused by mutations in the CNGB3 and CNGA3 genes, the Alachua, Florida, company said.

On Aug. 3, AGTC announced that the FDA granted orphan drug designation to the XLRP gene therapy candidate. The European Commission granted orphan medicinal product designation for the same indication in 2016.

XLRP is an inherited condition that causes progressive vision loss.

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