AGTC Files Investigational New Drug Application for the Treatment of Achromatopsia Caused by Mutations in the CNGA3 Gene

20:00 EDT 18 Oct 2016 | Globe Newswire

GAINESVILLE, Fla. and CAMBRIDGE, Mass., Oct. 19, 2016 (GLOBE NEWSWIRE) -- 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.

About Achromatopsia

Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind. The incidence rate for achromatopsia is approximately one in 30,000 people, and it is estimated that there are approximately 10,000 people in the United States and 17,000 people in Europe with achromatopsia.

About AGTC

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments.

AGTC's product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products.

Forward Looking Statements

This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others:  no gene therapy products have been approved in the United States and only two such products have been approved in Europe; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; uncertainty inherent in the regulatory review process; risks and uncertainties associated with drug development and commercialization, reliance on third parties over which AGTC may not always have full control; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2016, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.


David Carey (IR) or Danielle Lewis (PR)
Lazar Partners Ltd.
T: (212) 867-1768 or (212) 843-0211 or

Larry Bullock
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (386) 462-2204

Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 843-5775

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