AGTC to Present Preclinical Research Supporting Gene

GAINESVILLE, Fla. and CAMBRIDGE, Mass., May 02, 2022 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical-stage biotechnology company focused on the development of an adeno-associated virus (AAV)-based drug Gens is concentrating therapies to treat rare and debilitating diseases with an initial focus on inherited retinal disorders, preclinical data announced today, presented at the 25th Gens…th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), expanding the potential utility of its gene therapy platform to treat neurodegenerative diseases such as frontotemporal dementia, and to demonstrate robust gene expression using hybrid dual AAV vectors. The research will be presented in poster presentations at the in-person meeting, taking place May 16-19, 2022 in Washington, DC

“We are excited by the advances we have seen to date with our gene therapies in eye diseases and continue to evaluate the potential of our differentiated platform to treat a range of rare and debilitating diseases – including neurodegenerative diseases such as dementia,” said Dr Susan Schneider, Chief Medical Officers of AGTC. “We believe that the pre-clinical results in frontotemporal dementia are particularly encouraging and look forward to continuing our research to support a possible future filing for a new drug.”

AGTC Presentations at ASGCT 2022:

AVrh10-Based Gene Therapy for the Treatment of Frontotemporal Dementia Caused by GRN Mutations (Abstract #198)
Speaker: Dr. Khalid Arhzaouy, R&D Manager, AAV Gene Therapy, AGTC
Meeting Date/Time: May 16, 2022; 5:30 p.m. – 6:30 p.m. ET
Session Title: Gene Targeting and Gene Correction I
Poster No. M-79
Location: Hall D

  • In up to 10% of patients with frontotemporal dementia (FTD), the disease is caused by an inherited loss-of-function mutation in the granulin (GRN) gene and is characterized by a >50% reduction in progranulin, a highly conserved one primarily secreted protein expressed in the central nervous system (CNS).
  • In this study, progranulin is expressed in the CNS by an AAV vector encoding a human GREEN Gene packaged in AAVrh10 capsid and delivered directly into the cerebrospinal fluid via intracisternal magna injection (ICM).
  • Administration of AAVrh10-GRN to non-human primates resulted in dose-dependent and sustained expression of human progranulin in cerebrospinal fluid, reaching levels exceeding physiological levels in normal humans, without any vector-associated side effects.
  • The results support the feasibility of increasing progranulin expression by AAV-GRN gene therapy as a potential treatment for FDT caused by GREEN mutations.

Gene Therapy for Stargardt’s Disease Using Hybrid Dual AAV Vectors to Express ABCA4 (Abstract #306)
Moderator: Sharon Norton-Smith, Researcher, AGTC
Poster Session Date/Time: Monday, May 16, 2022; 5:30 p.m. – 6:30 p.m. ET
Session Title: Eye and Hearing Diseases
Poster No. M-187
Location: Hall D

  • Stargardt disease, the most common autosomal recessive form of early-onset macular dystrophy, is caused by mutations in the ABCA4 gene, which encodes the ATP-binding cassette transporter A4, which is expressed in the outer segments of photoreceptor cells in the retina.
  • This study evaluated a hybrid dual AAV strategy for in vivo Expression of ABCA4 to address potentially known packaging issues arising from the size of the ABCA4 gene.
  • The dual hybrid AAV vectors were capable of expressing both full-length ABCA4 proteins in vitro in HEK293 cells and in vivo in photoreceptor cells when administered by subretinal injection in both wild-type C57BL6 mice and the ABCA4 knock-out (K/O) mouse model.
  • Treatment of the ABCA4 K/O mice with the dual hybrid AAV system resulted in a reduction in toxic bisretinoids, and subretinal injection of the hybrid dual AAV vectors into non-human primates was safe and resulted in full expression of the ABCA4 protein length in the retina.

About AGTC
AGTC is a clinical-stage biotechnology company developing gene therapies for people with rare and debilitating diseases of the eyes, ears and central nervous system (CNS). AGTC is a leader in the development and engineering of all critical elements of gene therapy and bringing them together to create tailored therapies with the potential to address unmet patient needs. AGTC’s most advanced clinical programs leverage its world-class technology platform to improve the vision of patients with inherited retinal diseases. AGTC is conducting active clinical studies in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3). Its pre-clinical programs build on the company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing several key pipeline candidates to address significant unmet clinical needs in the areas of optogenetics, otology and CNS disorders and has formed 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 in neurotology. Visit for more information.

Forward-Looking Statements
This press release contains forward-looking statements that reflect AGTC’s plans, estimates, assumptions and beliefs, including statements regarding the potential of the Company’s gene therapy platform, ongoing preclinical development in frontotemporal dementia, potential clinical development in frontotemporal dementia and whether such work will be conducted will support future regulatory filings. Forward-looking statements include all statements that are not historical facts and are identified by words such as “anticipate,” “believe,” “may,” “aim,” “estimate,” “expect,” “intend,” “may,” , “plans”, “potential”, “forecast”, “projected”, “should”, “will”, “would” or similar expressions and the negative of these terms. Actual results could differ materially from those discussed in the forward-looking statements as a result of a number of important factors. Risks and uncertainties that could cause actual results to differ materially include, but are not limited to: gene therapy is new, with few treatments approved to date; AGTC cannot predict when or if it will receive regulatory approval to commercialize any product candidate or receive appropriate compensation; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; risks and uncertainties related to sources of funding for our development programs; the direct and indirect effects 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 included 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 at filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. In addition, forward-looking statements represent only management’s plans, estimates, assumptions and beliefs as of the date of this press release. Except as required by law, we undertake no obligation to publicly update these forward-looking statements or to update the reasons that actual results were material to us could differ from those anticipated in these forward-looking statements, even if new information becomes available in the future.

PR contact:
Kerry Sinclair
spectrum of science communication
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Company contact:
Jonathan dear
Applied Genetic Technologies Corporation
T: (617) 843-5778
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