NEW
YORK, May 7, 2024 /PRNewswire/ -- Myrtelle Inc.
("Myrtelle" or the "Company"), a clinical-stage gene therapy
company focused on developing transformative treatments for
neurodegenerative diseases, will host a symposium on Canavan
disease (CD) clinical trial updates and future directions for
oligodendrocyte targeting AAVs at the American Society of Gene and
Cell Therapy (ASGCT) 27th Annual Meeting on Wednesday, May 8, 2023, at 8:30 am EDT in Baltimore. The symposium will highlight
development of gene therapy targeting oligodendrocytes for Canavan
disease, updates on the company's First in Human (FIH) open-label
Phase 1/2 clinical trial using its proprietary vector
(rAAV-Olig001-ASPA), and the discovery of novel oligodendrocyte
targeting AAV capsids. Oligodendrocytes perform numerous key
functions in the brain - including the production of myelin, the
insulating material that enables proper neuronal function – and are
now appreciated as having roles in a range of diseases. Myrtelle is
developing a unique toolkit, including a proprietary class of
recombinant adeno-associated virus (rAAV) vectors that directly
target these cells thereby providing potential treatment options
for diseases in the company pipeline.
Symposium speakers include:
- Paola Leone, Ph.D., Professor
and Director of the Cell and Gene Therapy Center at Rowan-Virtua
SOM & School of Translational Biomedical Engineering &
Sciences Virtua Health College of Medicine and Life
Sciences of Rowan University will cover
the history of her work in Canavan disease in her talk on the
"Development of a Gene Therapy for Canavan Disease Targeting
Oligodendrocytes."
- Rob Lober, M.D. Ph.D. Principal
Investigator on Myrtelle's Canavan Disease Gene Therapy Trial and
Attending Neurosurgeon at Dayton Children's Hospital and Associate
Professor of Pediatrics at Wright State
University Boonshoft School of Medicine will present on a
case study from Myrtelle's Phase 1/2 First-in-Human Gene Therapy
Clinical Trial in Patients with Canavan Disease in his talk titled,
"Oligodendrocyte Targeted Gene Therapy for Canavan Disease."
- Patrick Aldrin-Kirk, Ph.D.
Chief Science Officer at rAAVen, an AAV engineering company focused
on generating innovative vectors for the next generation of gene
therapies, will present on the "Discovery of Novel Oligodendrocyte
Targeting AAV Capsids" and potential disease targets.
"As a leader in oligodendrocyte targeted therapies, we are
thrilled to have the opportunity to host a symposium at ASGCT's
annual conference," said Jordana
Holovach, Head of Communications and Community at Myrtelle.
"This symposium provides a unique forum to highlight the critical
but underappreciated role of oligodendrocytes in the overall health
of the brain and as a potential target for treating
neurodegenerative diseases. We are excited by the potential of our
evolving toolbox for delivering therapeutic constructs to
oligodendrocytes and impacting disease targets that are inherently
intractable by nature. We look forward to sharing our insights and
generating further interest into the oligodendrocyte story at the
symposium."
Date: Wednesday, May 8, 2024
Location: Baltimore Convention
Center – Room 324 – 326
Time: 8:30 am – 9:30 am
ABOUT MYRTELLE
Myrtelle Inc. is a gene therapy company focused on developing
transformative treatments for neurodegenerative diseases. The
company has a proprietary platform, intellectual property, and
portfolio of programs and technologies supporting innovative gene
therapy approaches for neurodegenerative diseases. Myrtelle has an
exclusive worldwide licensing agreement with Pfizer Inc. for its
Canavan disease program. For more information, please visit the
Company's website at: www.myrtellegtx.com.
ABOUT CANAVAN DISEASE
Canavan disease (CD) is a fatal childhood genetic brain disease
caused by mutations in the ASPA gene (ASPA) which prevent
the normal expression of aspartoacylase, a critical enzyme produced
in oligodendrocytes. The lack of normal aspartoacylase expression
negatively impacts brain development, including myelin production.
Patients with CD are impacted at birth but may appear normal until
several months old when symptoms begin to develop. Poor head
control, abnormally large head size, difficulty in eye tracking,
excessive irritability, severely diminished muscle tone, and delays
in reaching motor milestones, such as rolling, sitting, and
walking, are the typical initial manifestations of CD. As the
disease progresses, seizures, spasticity, difficulties in
swallowing, and overall muscle deterioration emerge with most
affected children developing life-threatening complications by
approximately 10 years of age. Currently, there are no cures for
CD, and only palliative treatments are available.
More information on Myrtelle's clinical trial in Canavan disease
can be found on https://clinicaltrials.gov/ under the
identifier NCT04833907 or by emailing
PatientAdvocacy@MyrtelleGTX.com.
Forward-Looking Statements
This press release contains forward-looking statements. Words
such as "may," "believe," "will," "expect," "plan," "anticipate,"
"estimate," "intend" and similar expressions (as well as other
words or expressions referencing future events, conditions, or
circumstances) are intended to identify forward-looking statements.
Forward-looking statements are based upon current estimates and
assumptions and include statements regarding results obtained to
date continuing to reinforce the hypothesis that addressing the
gene deficit directly in the brain, and specifically in
oligodendrocytes, of patients with Canavan disease can potentially
restore myelination and support functional improvements. While
Myrtelle believes these forward-looking statements are reasonable,
undue reliance should not be placed on any such forward-looking
statements, which are based on information available to us on the
date of this release. These forward-looking statements are subject
to various risks and uncertainties, many of which are difficult to
predict, that could cause actual results to differ materially from
current expectations and assumptions from those set forth or
implied by any forward-looking statements. Important factors that
could cause actual results to differ materially from current
expectations include, among others, Myrtelle's program
demonstrating safety and efficacy, as well as future results that
are consistent with prior results, the ability to generate the data
needed for further development of this novel gene therapy in the
patients with CD, and the ability to continue its clinical trials
and to complete them on time and achieve the desired results. All
forward-looking statements are based on Myrtelle's expectations and
assumptions as of the date of this press release. Actual results
may differ materially from these forward-looking statements. Except
as required by law, Myrtelle expressly disclaims any responsibility
to update any forward-looking statement contained herein, whether
as a result of new information, future events, or otherwise.
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SOURCE Myrtelle, Inc