Company Overview of Applied Genetic Technologies Corporation
Applied Genetic Technologies Corporation, a clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. The company’s lead products consist of four ophthalmology development programs across three targets, including X-linked retinoschisis that is in Phase I/II clinical trials; X-linked retinitis pigmentosa, which is in Phase I/II clinical trials; and achromatopsia that is in Phase I/II clinical trials for the treatment of inherited retinal diseases, as well as a preclinical program in adrenoleukodystrophy, which is a disease of the central nervous system and otology. It has collaboration agreements with Biogen M...
14193 NW 119th Terrace
Alachua, FL 32615
Founded in 1999
Key Executives for Applied Genetic Technologies Corporation
President, CEO & Director
Total Annual Compensation: $487.0K
VP & Chief Business Officer
Total Annual Compensation: $331.0K
Executive Director of Clinical Strategy
Total Annual Compensation: $400.0K
Chief Scientific Officer
Total Annual Compensation: $370.0K
Compensation as of Fiscal Year 2017.
Applied Genetic Technologies Corporation Key Developments
Applied Genetic Technologies Corporation Doses First Patient in Phase 1/2 Clinical Study of Gene Therapy for the Treatment of X-Linked Retinitis Pigmentosa
Apr 18 18
Applied Genetic Technologies Corporation announced that it has dosed the first patient in the Company’s Phase 1/2 clinical trial evaluating the safety and efficacy of an investigational AAV-based gene therapy for the treatment of X-linked retinitis pigmentosa. The multicenter study will assess AGTC’s novel recombinant AAV vector expressing a human RPGR gene in patients with XLRP. The patient is being followed by Dr. David Birch of Retina Foundation of the Southwest and the surgery was performed by Dr. Andreas Lauer of Oregon Health & Science University. The Phase 1/2 trial is an open-label, dose escalation study designed to assess the safety and efficacy of subretinal administration of the AAV-based gene therapy in approximately 15 patients diagnosed with XLRP. Trial participants will be enrolled sequentially in four groups. Individuals in Groups 1, 2 and 3 will receive a low, middle and high dose of the investigational study agent, respectively. Patients in Group 4 will receive the maximum tolerated dose as determined by the first three groups. The primary focus of the study will be to assess the safety of the vector through analysis of local (ocular) or systemic treatment-emergent adverse events. Efficacy will be measured by evaluation of changes in retinal structure, function and quality of life. XLRP is an inherited condition that causes progressive vision loss in boys and young men. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th decade of life. Preclinical data in a canine model of XLRP caused by mutations in the RPGR gene indicate that treatment with an AAV-based gene therapy product slowed the loss of visual function.
Applied Genetic Technologies Corporation Announces Completion of Enrollment of Phase 1 / 2 Clinical Study of Investigational Gene Therapy in Patients with X-Linked Retinoschisis
Apr 10 18
Applied Genetic Technologies Corporation announced the completion of enrollment in a clinical study of the company's gene therapy product candidate, in collaboration with Biogen, for the treatment of x-linked retinoschisis (XLRS). This multicenter study is designed to evaluate the collaboration’s AAV vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with XLRS caused by mutations in the RS1 gene. The Phase 1/2 trial is an open-label, dose escalation study designed to assess the safety and efficacy of intravitreal administration of the AAV-based gene therapy in approximately 27 patients diagnosed with XLRS caused by mutations in the RS1 gene. Trial participants were enrolled sequentially in four groups. Individuals in Groups 1, 2 and 3 received a low, middle or high dose of the investigational study agent. Patients in Group 4 received the maximum tolerated dose as determined by the first three groups. In addition, a group of pediatric patients were enrolled at the middle dose. Although the primary endpoint of this study is designed to evaluate safety, efficacy will also be measured by an evaluation of changes in visual structure, function and quality of life.
Applied Genetic Technologies Corporation Presents at ARM's 6th Cell & Gene Therapy Investor Day, Apr-17-2018 09:55 AM
Mar 28 18
Applied Genetic Technologies Corporation Presents at ARM's 6th Cell & Gene Therapy Investor Day, Apr-17-2018 09:55 AM. Venue: Metropolitan Club, One East 60th Street, New York, New York, United States. Speakers: Susan B. Washer, President, CEO & Director.
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