GAINESVILLE, Fla. and CAMBRIDGE, Mass., Sept. 28, 2021 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare inherited retinal diseases, today announced a presentation of the Company’s ongoing clinical trials in achromatopsia (ACHM) and X-linked retinitis pigmentosa (XLRP) at the Fourteenth International Symposium on Retinal Degeneration (RD2021).

Rachel Huckfeldt, MD, PhD, Assistant Professor of Ophthalmology at Harvard Medical School will present

Twelve-month Findings from Two Phase 1/2 Clinical Trials of Subretinal Gene Therapy for Achromatopsia

in adults and low-dose pediatrics on September 29, 2021, at 9:35 AM ET.

Paul Yang, MD, PhD, Assistant Professor of Ophthalmology at the Casey Eye Institute, Oregon Health & Science University in Portland, will present

Twelve-Month Analysis of Macular Structure using Optical Coherence Tomography (OCT) from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa

, on September 29, 2021, at 10:35 AM ET.

“We look forward to our investigators having the opportunity to share analysis of these results with members of the eye health community,” said Sue Washer, President and CEO of AGTC. “We believe the data provide important validation for the broad application of our AAV technology platform, including the best-in-class potential for our XLRP therapy candidate and further clinical investigation of our therapy to treat ACHM.”

“ACHM is an inherited condition caused by mutations in one of several genes, including the CNGB3 or CNGA3 genes, resulting in nonfunctioning “cone” photoreceptors responsible for color vision, and is associated with poor visual acuity, extreme light sensitivity leading to daytime blindness, and partial or complete loss of color discrimination,” said Dr. Huckfeldt. “These results are very encouraging, and I look forward to sharing them with my fellow retinal specialists.”

“Patients with XLRP experience damage to photoreceptors in the retina (both rods and cones) leading to declining vision in dim light conditions and progressive peripheral vision loss that can eventually result in legal blindness,” said Dr. Yang. “The sustained durability of improved visual function over 12 months is compelling evidence of biological activity for this XLRP gene therapy.”

AGTC plans to advance its ACHM program to the next stage of clinical development and is moving forward on an End-of-Phase 2 (EOP2) briefing packet to submit to the U.S. Food and Drug Administration, developing assays for pivotal ready testing, and planning production of clinical trial material. Along with the 12-month results in XLRP and other key XLRP data previously released by the company, AGTC is currently executing the Skyline and Vista Phase 2/3 trials that will expand safety and efficacy analyses, including microperimetry and luminance mobility maze outcomes.

Learn more and register here for the virtual/digital event to attend these important presentations: https://bit.ly/3z9uHre.

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 that address real patient needs. AGTC’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHMB3 and ACHMA3). 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 need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

Forward-Looking Statements


This release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements about the potential of the Company’s late-stage development programs in X-Linked Retinitis Pigmentosa (XLRP) and Achromatopsia (ACHM). Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to obtain the raw materials necessary to conduct its clinical trials. 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 or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our 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 our most recent annual report on Form 10-K and 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.

PR Contact:


Bryan Blatstein

Spectrum Science Communications

T: (212) 468-5379 or (917) 714-2609


[email protected]

Corporate Contact:


Stephen Potter

Chief Business Officer

Applied Genetic Technologies Corporation

T: (617) 413-2754


[email protected]