AGTC Presents Preclinical Research Supporting Gene Therapy Platform in Frontotemporal Dementia at 25th Annual Meeting of the American Society for Genetic and Cell Therapy

Applied Genetic Technologies Inc

Applied Genetic Technologies Inc

GAINESVILLE, Fla. and CAMBRIDGE, Mass. , May 02, 2022 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV) gene-based therapies to treat rare and debilitating diseases with an initial focus on retinal diseases Inherited, announced today preclinical data presented at 25y Annual meeting of the American Society for Genetic and Cell Therapy (ASGCT) that expands the potential usefulness of a gene therapy platform for treating obstetric neurodegenerative diseases, such as frontotemporal dementiaAnd and to demonstrate robust gene expression using dual-hybrid AAV vectors. Research on poster presentations will be presented at the in-person meeting May 16-19, 2022 in Washington, DC

Dr. said. Susan Schneider, AGTC’s medical director. “We believe the preclinical findings in frontotemporal dementia are particularly encouraging and we look forward to continuing our research to support a potential investigational new drug application in the future.”

AGTC offers at ASGCT 2022:

AVrh10-based gene therapy for frontotemporal dementia caused by GRN mutations (Abstract #198)
Presenter: Dr. Khaled Arzawi, Director of Research and Development, AAV Gene Therapy, AGTC
Session date/time: May 16, 2022; 5:30 PM – 6:30 PM ET
Session Title: Gene Targeting and Gene Correction 1
Poster No. M-79
Location: Hall D

  • In up to 10% of patients with frontotemporal dementia (FTD), the disease is caused by a loss-of-function genetic mutation in the granulin (GRN) gene, and is characterized by a >50% decrease in progranulin, a highly secreted protein that is constitutively conserved. . Expressed in the central nervous system (CNS).

  • In this study, progranulin is expressed in the central nervous system through the AAV transporter that encodes for human GRN The gene is packaged into the AAVrh10 capsid and delivered directly into the cerebrospinal fluid via intrauterine magna injection (ICM).

  • Administration of AAVrh10-GRN in non-human primates resulted in sustained dose-dependent expression of human progranulin in the cerebrospinal fluid and achieved levels above the physiological level in normal humans, without any vector-related adverse effects.

  • The results support the feasibility of increasing progranulin expression via AAV-GRN gene therapy as a potential treatment for .-induced FDT GRN mutations.

Gene therapy for Stargardt disease using dual-hybrid AAV vectors expressing ABCA4 (Abstract #306)
Presenter: Sharon Norton Smith, Researcher, AGTC
Poster session date/time: Monday, May 16, 2022; 5:30 PM – 6:30 PM ET
Session Title: Eye and Audiology Diseases
Poster No. M-187
Location: Hall D

  • Stargardt disease, the most common recessive form of early macular dystrophy, is caused by mutations in the ABCA4 gene that codes for the ATP-binding cassette transporter A4, expressed in the outer parts of the photoreceptor cells in the retina.

  • This study investigated the dual-hybrid AAV strategy for in vivo Expression of ABCA4 to address known packaging issues caused by the large size of the ABCA4 gene.

  • The double-hybrid AAV vectors were able to express both the full-length ABCA4 protein in the laboratory In HEK293 and . cells in vivo in photoreceptor cells when delivered by subretinal injection in both wild-type C57BL6 mice and in the ABCA4 knockout (K/O) mouse model.

  • Treatment of ABCA4 K/O mice with the dual-hybrid AAV system reduced toxic bisretinoids and subretinal injection of dual-hybrid AAV vectors into non-human primates was safe and resulted in expression of the full-length ABCA4 protein in the retina.

About AGTC
AGTC is a clinical-stage biotechnology company that develops genetic therapies for people with rare and debilitating eye, central nervous system and central nervous system (CNS) diseases. AGTC is a leader in the design, construction and combination of all critical elements of gene therapy to develop customized therapies with the potential to meet patients’ unmet needs. AGTC’s most advanced clinical programs leverage a best-in-class technology platform to improve vision for patients with genetic retinal diseases. The AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and color depth (ACHM CNGB3). Its preclinical programs are based on the company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC offers several critical candidates in pipelines to address significant unmet clinical needs in optogenetics, otology, and central nervous system disorders, and has entered into strategic partnerships with companies including Bionic Sight, an innovator in the field of optogenetics. Retina Coding and Otonomy, Inc. , a biopharmaceutical company dedicated to developing innovative neuroscience therapies. For more information, please visit the website

forward-looking statements
This release contains forward-looking statements that reflect the AGTC’s plans, estimates, assumptions, and beliefs, including statements about the potential of the company’s gene therapy platform, ongoing preclinical development in frontotemporal dementia, potential clinical development in frontotemporal dementia, and whether this work will support future regulatory filings. . Forward-looking statements include all statements that are not historical facts and can be identified by terms such as “expects,” “believes,” “could,” “seeks,” “estimates,” “expects,” “intends,” “plans,” “potential,” or “expects,” “projects,” “should,” “will,” “will,” 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 could cause actual results to differ materially include, among others: gene therapy is still new with few approved treatments yet; The AGTC cannot predict when or whether it will obtain regulatory approval to market a candidate product or receive reasonable compensation; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and marketing; risks and uncertainties related to the sources of financing for our development programmes; 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 under “Risk Factors” are contained in the company’s most recent annual report on Form 10-K, which may be supplemented by subsequent periodic reports filed with the SEC. In view of these uncertainties, you should not place undue reliance on these forward-looking statements. Also, the 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 publicly update these forward-looking statements or to update the reasons why actual results may differ materially from those projected in these forward-looking statements, even if new information becomes available in the future.

Public Relations Contact:
Keri Sinclair
Spectrum Science Communication

Company contact:
Jonathan Lieber
Chief Financial Officer
Applied Genetic Technologies Inc
Tel: 617-843-5778