AGTC Announces Publication of Data Supporting Further Investigation of Novel Potential Gene Therapy for Treating X-Linked Retinoschisis (XLRS) in Clinical Studies

GAINESVILLE, Fla. (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 eye diseases, today announced two studies supporting further clinical investigation of the Company's novel investigational gene therapy candidate for treating XLRS were published in Human Gene Therapy Clinical Development.

"These preclinical data are encouraging and support clinical studies of AAV-based gene therapies for use in vision-robbing retinal diseases," said Stephen M. Rose, Ph.D., Chief Research Officer, Foundation Fighting Blindness. "We are looking forward to seeing results from AGTC's ongoing clinical study and watching the Company's progress in bringing ocular gene therapies to market."

One study evaluated the safety and biodistribution profiles of rAAV2tYF-CB-hRS1, a recombinant AAV vector expressing retinoschisin (RS1), in RS1-deficient mice. Three groups of 20 mice each received an intravitreal injection in one eye of either vehicle or a higher or lower dose of vector. Results demonstrated that intravitreal injection of rAAV2tYF-CB-hRS1 was well tolerated in all groups with no test article-related changes in ophthalmic examinations. Microscopic pathology results demonstrated minimal to slight mononuclear cell infiltrates in 80 percent of vector-injected eyes at day 30, decreasing to 20 percent at day 90, with minimal ocular inflammatory cells detected by histopathology. RS1 expression, measured by immunohistochemistry, was demonstrated in all vector-injected eyes and was associated with decreased severity of splitting and disorganization of the inner nuclear layer of the retina at the higher dose level. Biodistribution studies demonstrated vector DNA in the injected eye and no vector DNA in any other tissue.

The other study evaluated the safety and biodistribution of the same AAV vector in three groups of normal male cynomolgus macaques (n=6 per group) administered either vehicle or a higher or lower dose of vector in one eye. Intravitreal administration of rAAV2tYF-CB-hRS1 was well-tolerated in all groups and was associated with dose-related anterior and posterior segment inflammatory responses that improved over time. There were no test article-related effects on intraocular pressure, electroretinography, visual evoked potential, hematology, coagulation, clinical chemistry or gross pathology observations. Microscopic pathology results demonstrated mononuclear cell infiltrates of minimal to moderate intensity in vector-injected eyes. RS1 expression, measured by immunohistochemistry, was seen in the retinal ganglion cell ring in all vector-injected eyes. Biodistribution studies demonstrated high levels of vector DNA in the injected eye with minimal or no vector DNA in other tissue.

"These preclinical studies support the further investigation of this novel AAV-based gene therapy candidate in clinical studies of patients with XLRS, an inherited retinal disease with no currently effective treatment," noted principal study investigator Jeff Chulay, M.D., DTM&H, Vice President and Chief Medical Officer. "In addition to significantly reduced visual acuity, XLRS can lead to serious complications including vitreous hemorrhage and retinal detachment. We are optimistic that the Company's technology, if approved, has the potential to improve the lives of patients worldwide suffering from this debilitating condition."

AGTC filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in March to conduct a Phase I/II clinical trial of the company's gene therapy product candidate for the treatment of XLRS. The XLRS clinical study is the first planned in a broad collaboration with Biogen focused on the development of gene-based therapies for orphan diseases of the retina. The collaboration includes three options for early stage discovery programs in two ophthalmic and one non-ophthalmic condition.

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 in ophthalmology. AGTC's lead product candidates focus on inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments.

AGTC has a robust product pipeline, including five named ophthalmology development programs across four targets (XLRS, XLRP, achromatopsia and wet age-related macular degeneration), one non-ophthalmology program (alpha-1 antitrypsin deficiency) and proof-of-concept data in multiple additional indications. The Company 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 expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation 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: uncertainty inherent in the regulatory review process; uncertainty regarding the ability to achieve the expected benefits from the proposed collaboration, including as a result of risks and uncertainties associated with drug development and commercialization, reliance on third parties over which AGTC may not always have full control and other risks associated with collaborations; and other risks and uncertainties described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2014, 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 Corporate Contact: Larry Bullock Chief Financial Officer Applied Genetic Technologies Corporation T: (386) 462-2204

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