AGTC to Present
Preclinical Research Supporting Gene Therapy Platform in Frontotemporal
Dementia at the 25th Annual Meeting of the American Society of Gene
& Cell Therapy
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
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 preclinical data presented at the 25th
Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
that expands the potential utility of its gene therapy platform to
address neurogenerative diseases, such as frontotemporal dementia, and to demonstrate robust gene expression using hybrid dual AAV vectors. The research will be featured in poster presentations at the in-person meeting taking place May 16-19, 2022 in Washington, D.C.
“We
are excited by the progress we’ve seen to date with our gene therapies
in ophthalmic diseases and are continuing to assess the potential of our
differentiated platform to treat a range of rare and debilitating
diseases – including neurodegenerative diseases like dementia,” said Dr.
Susan Schneider, Chief Medical Officer of AGTC. “We believe the
preclinical findings in frontotemporal dementia are particularly
encouraging and look forward to continuing our research to support a
potential future Investigational New Drug Application filing.”
AGTC presentations at ASGCT 2022:
AVrh10-Based Gene Therapy for the Treatment of Frontotemporal Dementia Caused by GRN Mutations (Abstract #198) Presenter: Dr. Khalid Arhzaouy, R&D Manager, AAV Gene Therapy, AGTC Session Date/Time: May 16, 2022; 5:30 p.m. – 6:30 p.m. ET Session Title: Gene Targeting and Gene Correction I Poster #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 marked by >50% reduction in progranulin, a highly conserved
secreted protein primarily expressed in the central nervous system
(CNS).
In this study, progranulin is expressed in the CNS through an AAV vector encoding a human GRN gene packaged in AAVrh10 capsid and delivered directly to cerebrospinal fluid via intracisternal magna (ICM) injection.
Administration
of AAVrh10-GRN in non-human primates resulted in a dose-dependent and
sustained expression of human progranulin in cerebrospinal fluid and
achieved levels above the physiological level in normal humans, without
any vector-associated adverse effects.
Results support the
feasibility of augmenting progranulin expression via AAV-GRN gene
therapy as a potential treatment for FDT caused by GRN mutations.
Gene Therapy for Stargardt Disease Using Hybrid Dual AAV Vectors to Express ABCA4 (Abstract #306) Presenter: Sharon Norton-Smith, Researcher, AGTC Poster Session Date/Time: Monday, May 16, 2022; 5:30 p.m. – 6:30 p.m. ET Session Title: Ophthalmic and Auditory Diseases Poster # 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 that codes
for ATP-binding cassette transporter A4, expressed in the outer segments
of photoreceptor cells in the retina.
This study investigated a hybrid dual AAV strategy for in vivo expression of ABCA4 to potentially address known packaging issues resulting from the large size of the ABCA4 gene.
The hybrid dual AAV vectors were able to express full length ABCA4 protein both in vitro in HEK293 cells and in vivo
in photoreceptor cells when delivered by subretinal injection in both
wild type C57BL6 mice and in the ABCA4 knock-out (K/O) mouse model.
Treatment
of the ABCA4 K/O mice with the hybrid dual AAV system led to reduced
toxic bisretinoids and subretinal injection of the hybrid dual AAV
vectors in non-human primates was safe and resulted in expression of
full-length ABCA4 protein in the retina.
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 X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM
CNGB3). 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 https://agtc.com/.
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 gene therapy platform, the ongoing
preclinical development in frontotemporal dementia , the potential
clinical development in frontotemporal dementia, and whether such 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 "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;
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.
BioFlorida is the voice of Florida's life sciences industry, representing 8,600 establishments and research organizations in the BioPharma, MedTech, Digital Health and Health Systems that collectively employ nearly 107,000 Floridians. Source: TEConomy/BIO (released 2022)
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