Applied Genetic Technologies, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, has filed an Investigational New Drug application (IND) with the FDA to conduct a phase I/II 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. 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, to develop gene-based therapies for multiple ophthalmic diseases.
“We are deeply committed to advancing therapies to address the unmet needs of patients with rare inherited retinal disorders, and filing our fifth IND in this field represents a significant milestone that may ultimately improve the lives of individuals affected by XLRP,” said Sue Washer, President and CEO of AGTC. “Gene therapy clinical trials continue to rapidly progress, making this an exciting time for AGTC, the clinical community and patients affected by ocular conditions with no currently available treatments.”
AGTC 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 United States in the coming months.
AGTC is currently scheduling patients for enrollment in clinical trials for XLRS as well as for achromatopsia caused by mutations in the CNGB3 and CNGA3 genes.