"We are encouraged by these study results that further underscore the safety of novel AAV-based gene therapies to treat rare inherited eye diseases with few currently available therapeutic options," said
Title: Safety and Biodistribution Study of rAAV2tYF-CB-hRS1 in RS1-deficient Mice
Abstract #: 350775
This study evaluated the toxicology and biodistribution profiles of a recombinant adeno-associated virus (rAAV) vector expressing the human retinoschisin (hRS1) gene at two doses in three groups of male RS1-deficient mice. Results demonstrated that intravitreal injection of rAAV2tYF-CB-hRS1 was well tolerated with minimal ocular inflammatory cells detected by histopathology. RS1 expression, measured by immunohistochemistry, was associated with decreased severity of splitting and disorganization of the inner nuclear layer of the retina at the higher dose level, supporting the use of this vector in patients with XLRS.
Title: Safety and Biodistribution Study of rAAV2tYF-CB-hRS1 in Nonhuman Primates
Abstract #: 350780
This study evaluated the toxicology and biodistribution of an AAV vector expressing retinoschisin in male cynomolgus macaques. Results demonstrated that administration of rAAV2tYF-CB-hRS1 in normal cynomolgus macaques was associated with dose-related anterior and posterior segment inflammatory response that improved over time. Histological examination revealed that mononuclear cell infiltrates of minimal to moderate intensity were more common in the higher dose group and RS1 expression in the retinal ganglion cell ring was demonstrated by immunohistochemistry. These results further support the use of rAAV2tYF-CB-hRS1 in clinical studies of patients with XLRS.
Title: Initial Safety Evaluation of rAAV-hCNGB3 Vectors in Nonhuman Primates
Abstract #: 350277
Previous studies in CNGB3-mutant dogs have shown that subretinal injection of an AAV5 vector expressing a human CNGB3 gene can rescue the ACHM electroretinography (ERG) phenotype, but also showed that at high doses, many of the animals developed chorioretinitis, a toxicity consistent with an immune response to a foreign protein. This study evaluated eight cynomolgus macaques that received subretinal injections of AAV2tYF-hCNGB3 or AAV5-hCNGB3 vectors, followed by ophthalmic examinations, digital fundus photography, scotopic and photopic ERG and visual evoked potentials (VEP) and histological examination. Results showed that both vectors were well tolerated and were associated with dose-related ocular inflammation that was milder than the severe inflammation seen in dogs receiving high dose of AAV5-hCNGB3. No test article-related effects on ERG or VEP were observed. These findings will be helpful for guiding future development of rAAV-CNGB3 gene therapies for humans.
Title: Efficient Clearance of Herpes Simplex Virus Using a GMP-compliant Method for Production of Recombinant Adeno-associated Virus Vectors
Abstract #: 350887
This study evaluated the safety of AGTC's proprietary, herpes simplex virus (HSV) manufacturing process for production of recombinant AAV virus vectors. Results demonstrated that the company's process to produce and purify rAAV vectors for use in patients with XLRS and ACHM effectively inactivates and removes HSV particles during vector production. The study authors concluded that the risk of HSV-related adverse events in patients receiving rAAV vectors purified in this manner is very low.
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 X-linked retinoschisis, achromatopsia and X-linked retinitis pigmentosa, which are inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC is also using its gene therapy expertise to expand into disease indications with large market opportunity such as wet AMD.
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, including,
without limitation, those described under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended
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