Applied Genetic Technologies (AGTC) Files IND for Treatment of Achromatopsia Caused by Mutations in CNGA3 Gene

October 19, 2016 4:44 PM EDT
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Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration to conduct a Phase I/II clinical trial of the Company's gene therapy product candidate for the treatment of achromatopsia caused by mutations in the CNGA3 gene. The Company previously initiated a Phase I/II clinical trial of its gene therapy product for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

"Achromatopsia severely impairs vision of affected individuals which significantly impacts their daily activities,” said Sue Washer, President and CEO. “The filing of an IND for our second achromatopsia therapeutic candidate reflects our commitment to advancing therapies that address the unmet needs of patients with the two mutations that are the most common causes of this disease. We believe this filing validates the flexibility and broad utility of our AAV-based gene therapy platform to develop multiple gene-based therapies for rare, inherited ophthalmic diseases and this program will be the fourth ophthalmic gene therapy candidate we have advanced to human trials.”

Achromatopsia results from mutations in one of several genes. Two of these genes, CNGA3 and CNGB3, encode the alpha and beta subunits, respectively, of an ion channel that is essential for proper function of cone cells within the retina. About 75 percent of achromatopsia patients have mutations in one of these two genes; the remainder of cases result from mutations in one of several other genes. Preclinical studies in dog and sheep models of achromatopsia demonstrate that the animals are better able to navigate a maze after receiving gene therapy that delivers a functional copy of CNGA3 or CNGB3.

The Company plans to initiate a clinical study evaluating the safety and efficacy of its proprietary gene therapy for CNGA3-related achromatopsia in the United States in the coming months.



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