May 27, 2017 3:01 AM ET

Biotechnology

Company Overview of Applied Genetic Technologies Corporation

Company Overview

Applied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops genetic therapies to treat patients with inherited diseases. Its lead products consist of four ophthalmology development programs across three targets, including X linked retinoschisis (XLRS) that is in a Phase I/II clinical trials for the treatment of an inherited retinal disease; X-linked retinitis pigmentosa (XLRP), which is enrolling patients in a Phase I/II clinical trials; and achromatopsia (ACHM) that is enrolling patients in a Phase I/II clinical trials. Applied Genetic Technologies Corporation has collaboration agreements with Biogen MA, Inc.; 4D Molecular Therapeutics; Synpromics Limited; and...

14193 NW 119th Terrace

Suite 10

Alachua, FL 32615

United States

Founded in 1999

53 Employees

Phone:

386-462-2204

Key Executives for Applied Genetic Technologies Corporation

Chief Executive Officer, President and Director
Age: 55
Total Annual Compensation: $464.0K
Chief Financial Officer
Age: 60
Total Annual Compensation: $327.0K
Chief Business Officer and Vice President
Age: 60
Total Annual Compensation: $314.2K
Executive Director of Clinical Strategy
Age: 69
Total Annual Compensation: $381.5K
Chief Scientific Officer
Age: 56
Total Annual Compensation: $400.0K
Compensation as of Fiscal Year 2016.

Applied Genetic Technologies Corporation Key Developments

Applied Genetic Technologies Corporation Presents New Data on its AAV-Based Gene Therapies for the Treatment of Achromatopsia and X-Linked Retinitis Pigmentosa at the American Society of Gene and Cell Therapy Annual Meeting

Applied Genetic Technologies Corporation announced the presentation of new data from studies in animal models of achromatopsia (ACHM) and X-linked retinitis pigmentosa (XLRP) that support the company’s clinical development programs in these indications. The data were presented at the American Society of Gene and Cell Therapy 20th Annual Meeting, taking place in Washington, D.C., May 10-13. ACHM and XLRP are rare inherited retinal diseases. ACHM results from mutations in either of the CNGB3 or CNGA3 genes. Mutations in these genes account for approximately 75% of the total achromatopsia patient population. Individuals with achromatopsia have markedly reduced visual acuity, extreme light sensitivity, and complete loss of color discrimination. XLRP affects boys, causing night blindness by the time they are ten, and progresses to legal blindness by their early forties. AGTC is developing a gene-based therapy for XLRP in collaboration with Biogen and expects to file an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration for this product candidate this year. No systemic toxicity was associated with treatment and no consistent test article-related effects were observed. Two out of five animals treated with the higher dose of AAV2tYF-PR1.7-CNGA3 had microscopic findings of outer retinal atrophy, with or without inflammatory cells in the retina and choroid that were considered procedural- and/or test article-related. All vector-treated eyes demonstrated CNGA3 expression, and developed cone-mediated electroretinogram (ERG) responses with no change in rod-mediated ERG responses. Improvements in maze navigation times and obstacle collisions were observed in all vector-treated eyes compared with control eyes and with pre-dose results in the treated eyes. The researchers conclude that these results support the use of AAV2tYF-PR1.7-hCNGA3 in clinical studies in patients with achromatopsia caused by mutations in CNGA3. The poster will include results from a study evaluating the efficacy of two vectors (AAV2tYF-GRK1-RPGRco and AAV2tYF-GRK1-RPGRstb) containing the AAV2tYF capsid, human GRK1 promoter and a codon-optimized or stabilized version of the human RPGR gene administered subretinally in an animal model of mid-stage XLRP resulting from mutations in the RPGR gene. In this model, mid-stage disease occurs when animals are approximately 12 weeks of age and is associated with an approximate 40% loss of photoreceptors. Two animals per group received RPGRco in the right eye and RPGRstb in the left eye at each of three dose levels. Rescue of photoreceptor structure was assessed by clinical examination and histology and/or immunohistochemistry on retinal cryosections eight weeks post injection. No abnormal ophthalmic findings were noted in any eyes at the middle- or low-dose levels. Fundoscopic examination at 8 weeks post-dosage showed signs of retinal detachment and inflammation in the eyes injected with the high dose of either RPGRco or RPGRstb. Dose-dependent RPGR transgene expression was observed with both vectors, with greater RPGR expression noted in eyes injected with RPGRco compared with contralateral eyes injected with RPGRstb at the same dose levels. Correction of rod opsin and middle/long wavelength cone opsin mislocalization was demonstrated in all AAV-RPGR treated eyes. Researchers conclude that the results demonstrate greater RPGR expression with RPGRco compared with RPGRstb, and that the middle doses of both vectors resulted in optimal correction at mid-stage disease with limited inflammation in this animal model of XLRP.

Applied Genetic Technologies Corporation to Report Q3, 2017 Results on May 10, 2017

Applied Genetic Technologies Corporation announced that they will report Q3, 2017 results at 5:00 PM, Eastern Standard Time on May 10, 2017

Applied Genetic Technologies Corporation, Q3 2017 Earnings Call, May 10, 2017

Applied Genetic Technologies Corporation, Q3 2017 Earnings Call, May 10, 2017

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