Company initiates partnership and
collaboration with UMass Chan Medical School to characterize
relevant targets that enable ASO-mediated correction for
FXS
Fragile X syndrome is a rare, genetic
neurological disease that is the leading inherited form
of intellectual disability and a known cause of
autism
CAMBRIDGE, Mass., June 25,
2024 /PRNewswire/ -- QurAlis Corporation
("QurAlis"), a clinical-stage biotechnology company driving
scientific breakthroughs into powerful precision medicines that
have the potential to alter the trajectory of amyotrophic
lateral sclerosis (ALS), frontotemporal dementia (FTD), and
other neurodegenerative and neurological diseases, today
announced it is expanding its industry-leading precision medicine
antisense oligonucleotide (ASO) splicing expertise beyond
neurodegeneration into Fragile X syndrome (FXS).
As part of this strategy, QurAlis has initiated a partnership
and collaboration with UMass Chan Medical School to explore the
biology of FXS to determine and confirm relevant targets that could
enable ASO-mediated correction for FXS. QurAlis can leverage its
deep understanding, knowledge and expertise in developing ASOs
including its proprietary FlexASO™ Splice Modulator Platform as
part of the collaboration.
"QurAlis' deep knowledge and expertise of ASO splicing targets
combined with our FlexASO™ Splice Modulator Platform gives us
the latitude to go beyond neurodegeneration to explore the
potential of bringing much-needed precision medicines for other
serious neurological diseases like FXS," said Kasper Roet, Ph.D., chief executive officer and
co-founder of QurAlis. "Our partnership and collaboration with
UMass Chan in FXS underscores the enormous value of partnerships
between academia and industry to drive innovation forward to help
fulfill unmet medical needs for patients. We are excited to expand
our scope into a new therapeutic area and to make use of our
expertise in splicing targets so that QurAlis can continue the goal
of making a real difference in patients' lives."
"We are excited to be partnering with QurAlis in the next step
of this years-long research," said Joel
Richter, Ph.D., the Arthur F. Koskinas Chair in Neuroscience
and professor of molecular medicine at UMass Chan Medical School in
Worcester, MA. "QurAlis'
translational knowledge and clinical trial experience in ASO
splicing for neurodegenerative disorders is an important component
to bridging the gap between biomedical discoveries made in the
laboratory and delivering therapies to patients in the clinic. With
their help, we are hopeful that one day we may be able to offer
patients with FXS meaningful treatment options."
About Fragile X Syndrome (FXS)
Fragile X syndrome is
the leading inherited form of intellectual disability and a known
cause of autism. It is a genetic condition caused by a mutation of
a single gene – FMR1 – on the X chromosome. This mutation of FMR1
causes a range of developmental problems including learning
disabilities, behavioral challenges, and cognitive impairment.
An orphan disease, FXS affects approximately 87,000 individuals
in the U.S. alone – one in 4,000 men and one in 6,000 women. Though
FXS occurs in both genders, males are more frequently affected than
females, and generally with greater severity. In addition to
intellectual disability, FXS patients endure a wide range of
disabling symptoms including severe anxiety, social aversion,
hyperactivity and attention deficit, sensory hypersensitivity,
aggression, developmental seizures, and others. There are no
disease-modifying therapies currently available for FXS.
About QurAlis' FlexASO™ Splice Modulator
Platform
Antisense oligonucleotides (ASOs) are short,
engineered single-stranded DNA/RNA molecules that can selectively
bind RNA to regulate its expression in the cell. ASO technology has
been leading in the field of protein regulation and has since
allowed us to develop treatments for neurodegenerative disease by
changing the expression of genes connected to the
disease.
QurAlis' FlexASO™ Splice Modulator Platform was
developed to generate splice-switching ASOs with improved potency,
increased therapeutic index and improved bio-distribution. This
bespoke platform has the potential to tackle the spectrum of
neurodegenerative and neurological diseases.
About QurAlis Corporation
At QurAlis, we are neuro
pioneers on a quest to cure. We work with a relentless pursuit of
knowledge, a precise attention to craft, and an optimistic mindset
to discover and develop effective precision medicines that have the
potential to alter the trajectory of amyotrophic lateral sclerosis
(ALS), frontotemporal dementia (FTD), and other neurodegenerative
and neurological diseases. Founded by an internationally recognized
team of neurodegenerative biologists from Harvard Medical School and Harvard University, QurAlis is advancing a robust
precision medicine pipeline with therapeutic candidates aimed at
modifying severe disease pathology in defined patient populations
based on both disease-causing genetic mutation(s) and clinical
biomarkers. For more information, please visit www.quralis.com or
follow us on X @QurAlisCo or LinkedIn.
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SOURCE QurAlis