![]() AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies with the potential to address unmet patient needs. In general, XLRP patients experience a gradual decline in visual acuity over the disease course, which results in legal blindness around the 4th or 5th decade of life.ĪCHM is an inherited condition associated with extremely poor visual acuity (most affected individuals are legally blind), extreme light sensitivity resulting in daytime blindness, and complete loss of color discrimination.ĪGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field. XLRP is an inherited condition that causes progressive vision loss in boys and young men. Presentation Date/Time: Paper on Demand, recorded presentation available to attendees during and after the ASRS meetingĪdditional information can be found on the 40 th Annual Scientific Meeting schedule.Ībout X-Linked Retinitis Pigmentosa (XLRP) Presenter: Rajiv Anand, MD, FRCS, FASRS, Texas Retina Associates, Dallas, Texas Sub-retinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Phase 2 Randomized, Controlled, Masked Multicenter Clinical Trial (Skyline) Interim Safety Results Presentation Date/Time: J3:57 PM – 4:03 PM ET Presenter: Lejla Vajzovic, MD, FASRS, Assistant Professor of Ophthalmology, Adult and Pediatric Vitreoretinal Surgery and Diseases, Duke University Eye Center, Durham, NC Interim Subretinal Gene Therapy Safety Results in Two Phase 1/2 Open-label, Dose-escalation Clinical Trials to Treat Achromatopsia “We look forward to sharing future updates as we continue to progress these assets through their respective clinical development paths.” ![]() Susan Schneider, Chief Medical Officer of AGTC. ![]() ![]() “We are honored to join our colleagues in the ophthalmology community, during this important retina meeting, to present data from the exciting gene therapies we are developing at AGTC as potential treatments for both ACHM and XLRP,” said Dr. Each presentation will be shared during the American Society of Retina Specialists (ASRS) Annual Meeting, taking place July 13-16 in New York City. The other presentation will focus on interim safety results in the ongoing Phase 2 Skyline trial of AGTC-501, a recombinant AAV viral vector-based gene therapy targeting mutations in the RPGR gene in patients with X-linked retinitis pigmentosa (XLRP). One of the presentations will focus on interim safety results in two Phase 1/2 dose-escalation clinical trials of AGTC-401 and AGTC-402 in patients with achromatopsia (ACHM) caused by mutations in the CNGB3 and CNGBA3 genes respectively. GAINESVILLE, Fla., and CAMBRIDGE, Mass., J(GLOBE NEWSWIRE) - Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical-stage biotechnology company focused on the development of adeno-associated virus (AAV)-based gene therapies for the treatment of rare and debilitating diseases with an initial focus on inherited retinal diseases, today announced two encore presentations on the interim safety results in three studies.
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