GAINESVILLE, Fla., and CAMBRIDGE, Mass., July 07, 2022 (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. 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. 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). Each presentation will be shared during the American Society of Retina Specialists (ASRS) Annual Meeting, taking place July 13-16 in New York City.

“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. Susan Schneider, Chief Medical Officer of AGTC. “We look forward to sharing future updates as we continue to progress these assets through their respective clinical development paths.”

Presentation details are as follows:

Interim Subretinal Gene Therapy Safety Results in Two Phase 1/2 Open-label, Dose-escalation Clinical Trials to Treat Achromatopsia


Presenter: Lejla Vajzovic, MD, FASRS, Assistant Professor of Ophthalmology, Adult and Pediatric Vitreoretinal Surgery and Diseases, Duke University Eye Center, Durham, NC

Session Date/Time: July 16, 2022; 3:35 PM – 4:15 PM ET

Presentation Date/Time: July 16, 2022; 3:57 PM – 4:03 PM ET

Sub-retinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Phase 2 Randomized, Controlled, Masked Multicenter Clinical Trial (Skyline) Interim Safety Results


Presenter: Rajiv Anand, MD, FRCS, FASRS, Texas Retina Associates, Dallas, Texas

Presentation Date/Time: Paper on Demand, recorded presentation available to attendees during and after the ASRS meeting

Additional information can be found on the 40

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Annual Scientific Meeting schedule.

About X-Linked Retinitis Pigmentosa (XLRP)

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 or 5th decade of life.

About Achromatopsia (ACHM)

ACHM 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.

About AGTC

AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. 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. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases. AGTC has active clinical trials in XLRP and ACHM. Its preclinical programs build on the company’s industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical needs in optogenetics, otology and CNS disorders, and has entered strategic collaborations with companies including Bionic Sight, an innovator in the emerging field of optogenetics, and retinal coding and Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. For more information, please visit

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Forward-Looking Statements


This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the safety of AGTC-401, AGTC-402, and AGTC 501, the potential for the clinical development of AGTC-401 as a treatment of ACHM and AGTC-501 as a treatment for XLRP, the continued safety development profile of AGTC-401 and AGTC-501, the ability to use the interim results clinical study results for AGTC-401 and AGTC-501 as a predictor of success, and whether these results will support future regulatory filings for AGTC-401 and AGTC-501. 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: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate, including but not limited to XLRP, or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; risks and uncertainties related to funding sources for our development programs; the direct and indirect impacts of the ongoing COVID-19 pandemic on the Company’s business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading “Risk Factors” in the company’s most recent annual report on Form 10-K, as it may be supplemented by subsequent periodic reports 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.

Corporate Contact:

Jonathan Lieber

Chief Financial Officer

Applied Genetic Technologies Corporation

T: (617) 843-5778

[email protected]