ATLANTA, June 19, 2024 /PRNewswire/ -- The CMT
Research Foundation, a non-profit focused solely on delivering
treatments and cures for Charcot-Marie-Tooth disease (CMT)*, has
formed a strategic partnership with ReviR Therapeutics to
advance small molecule therapeutics that modulate the expression of
the gene that causes CMT1A.
ReviR Therapeutics is developing a class of drugs called
small molecule splice modulators, which can regulate the expression
of a gene by influencing which components of the genetic code get
included in the final version of the RNA transcript. In this
project supported by the CMTRF, ReviR Therapeutics will
develop a splice modulator that will reduce the expression of the
disease gene, which is overexpressed in CMT1A and is the cause of
disease symptoms. A benefit of this type of drug is that it can be
taken orally, making it much more convenient for patients compared
to the injections required for other approaches. The technologies
and approaches from ReviR Therapeutics could
revolutionize treatment paradigms not only for CMT1A but
potentially for other forms of CMT and other neurodegenerative
disorders as well.
"Our platform technology at ReviR represents a radical
departure from traditional genetic therapies that often require
invasive administration methods," says Peng
Yue, Ph.D., Chief Executive Officer and Co-founder
of ReviR Therapeutics. "We have designed small molecules that
can be taken orally, cross the blood-nerve barrier, and directly
interact with mRNA to modulate splicing. The splicing change will
cause mRNA destruction and will effectively reduce disease-gene
protein levels to address the genetic basis of CMT1A."
"Collaborating with ReviR Therapeutics aligns
perfectly with CMTRF's mission to accelerate the development of
therapies that can halt or even reverse the progression of CMT,"
says Cleary Simpson, CEO of the
CMTRF. "Their cutting-edge technology has the potential to
transform the lives of those living with this chronic condition by
providing a non-invasive, potent, and easily administrable
treatment option."
The experiments in this project will validate the effectiveness
of these splicing modulators in cell models of CMT1A, with the goal
to quickly advance to animal studies and, eventually, clinical
trials.
"We are deeply committed to this research program and I've
spent the last decade examining new approaches to treat CMT
patients safely and effectively," Dr. Paul
August, Chief Scientific Officer
of ReviR Therapeutics and who serves on CMTRF's
scientific advisory board, adds, "Our goal is not just to innovate
but to apply our innovations in ways that offer real, tangible
benefits to patients. By reducing disease-gene protein levels with
an orally administered drug, we hope to provide a therapy that is
both effective and aligns with everyday life, minimizing the
psychological and physical toll of CMT1A."
"ReviR Therapeutics has developed a groundbreaking type of
medication called SpliceR, which belongs to a new category of
genetic treatments. These treatments work by tweaking the process
of how genetic information is used to make proteins, a process
somewhat similar to the way the FDA-approved
drug Risdiplam treats spinal muscular
atrophy. Risdiplam helps by adjusting a gene to produce a
vital protein needed by the body," explains Dr. August.
"Similarly, ReviR's SpliceR drugs make
adjustments to the genetic information but in a different
way. SpliceR drugs add a piece to the genetic message that
tells the body to stop expressing the protein and the position of
this stop signal leads to the reduction of problematic proteins.
This approach could be a big step forward compared to some other
gene therapies, making treatment less burdensome and more
accessible."
ReviR Therapeutics is an emerging biopharmaceutical
company focused on the development of RNA-targeting small molecule
therapies that modulate the causal genes of CNS diseases, cancer,
and other rare genetically defined diseases. The company is
utilizing cutting-edge technologies to develop therapies with the
goal of developing disease modifying therapies that are
efficacious, safe, and specific. The company's initial focus is to
apply SpliceR modulation technologies towards RNA targets of
genetic diseases with limited or no treatment options. ReviR
Therapeutics was founded in 2021 and headquartered in Brisbane, CA.
CMT Research Foundation (CMTRF) is a patient-led,
non-profit focused on delivering treatments and cures for CMT. The
foundation identifies significant obstacles or deficiencies
impeding progress toward a cure and seeks out collaborators to
address these issues. To date, CMTRF has funded 24 projects, of
which 8 are completed. Of those 8 completed projects, 5 have
clinical candidates. CMTRF's mission to invest in promising science
with high potential of leading to treatments and cures was proven
effective and ground-breaking when DTx Pharma with a
CMTRF- backed program as its lead candidate was acquired by
Novartis for $1 billion. Founded by
two patients who are driven to expedite drug delivery to people who
live with CMT, the 501(c)(3) federal tax-exempt organization is
supported by personal and corporate financial gifts.
*Charcot-Marie-Tooth encompasses a group of
inherited, chronic peripheral neuropathies that result in nerve
degradation. CMT patients suffer from progressive muscle atrophy of
legs and arms, causing walking, running and balance problems as
well as abnormal functioning of hands and feet. CMT affects one in
2,500 people (about the same prevalence as cystic fibrosis),
including 150,000 Americans and nearly 3 million people
worldwide. At the moment, there is no treatment or cure for
CMT.
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SOURCE ReviR Therapeutics