SUZHOU, China, July 25,
2024 /PRNewswire/ -- Ractigen Therapeutics, a
pioneering developer of small activating RNA (saRNA) therapeutics,
today announced that the U.S. Food and Drug Administration (FDA)
has granted Rare Pediatric Disease Designation (RPDD) to RAG-18,
one of the company's lead saRNA product candidates for the
treatment of Duchenne Muscular Dystrophy (DMD). RAG-18 could
represent a novel and translatable therapeutic strategy for DMD and
Becker Muscular Dystrophy (BMD) caused by any mutation of the DMD
gene.
The RPDD is granted by the FDA to drugs that treat serious or
life-threatening diseases primarily affecting children under 18
years of age with a prevalence of fewer than 200,000 in
the United States. If a New Drug
Application (NDA) for RAG-18 is approved, Ractigen may be eligible
to receive a Priority Review Voucher (PRV), which can be used to
expedite the review of a subsequent marketing application for a
different product or sold to another company with historical
transaction values exceeding $100
million and reaching as high as $350
million, representing a substantial financial opportunity
for Ractigen.
Dr. Long-Cheng Li, Founder and
CEO of Ractigen Therapeutics, stated: "Receiving the RPDD for
RAG-18 is a milestone for Ractigen and reinforces our commitment to
developing innovative therapies for rare diseases. This designation
not only accelerates the development of RAG-18 but also opens
opportunities for future advancements in RNAa therapies. We are
dedicated to improving the lives of patients with DMD and other
rare diseases."
About RAG-18
RAG-18 is a first of its kind saRNA candidate designed to
specifically target and activate UTRN gene expression in muscle
cells via RNAa mechanism. The utrophin protein encoded by the UTRN
gene is structurally and functionally similar to dystrophin, and
its upregulation could potentially serve as a functional
replacement for the missing dystrophin in DMD muscle cells,
providing treatment for all DMD patients regardless of the specific
mutation location. Preclinical data indicate that RAG-18, delivered
through subcutaneous injection utilizing Ractigen's proprietary
LiCOTM (lipid-conjugated oligonucleotide) technology,
has effectively mitigated muscle damage, demonstrating significant
potential in treating DMD patients..
About DMD
Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy
(BMD) are severe genetic disorders caused by mutations in the
dystrophin gene, leading to the absence or insufficiency of
functional dystrophin protein. This protein is essential for muscle
fiber stability. Without it, muscle cells are easily damaged and
cannot repair themselves, resulting in progressive muscle weakness
and degeneration. The dystrophin gene, the largest in the human
body, contains 79 exons. Current disease-modifying therapeutic
approaches for DMD include antisense oligonucleotides (ASO)
mediated exon skipping, gene therapy, and gene editing, with exon
skipping being the most common strategy. However, these treatments
have significant limitations, highlighting the critical need for
innovative therapies that target the root cause of DMD to provide
more effective and long-lasting benefits for patients.
About RNAa
RNAa is a clinically validated platform technology developed by
Dr. Long-Cheng Li and his team. It
utilizes saRNAs to target gene regulatory domains, activating gene
expression and restoring therapeutic protein levels. This
innovative technology holds vast potential for developing
therapeutic drugs across various diseases, particularly where
traditional methods fall short, including genetic disorders.
About Ractigen Therapeutics
A leader in saRNA drug development, Ractigen Therapeutics is at
the forefront of developing saRNA drugs utilizing the RNAa
mechanism to up-regulate endogenous gene expression. This
innovative approach involves saRNA targeting specific genes to
enhance transcription, thereby restoring normal protein functions.
Ractigen's cutting-edge technology is pivotal in treating diseases
unaddressable by conventional methods, such as those resulting from
epigenetic silencing or gene downregulation. For more
information, please visit our website at www.ractigen.com.
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SOURCE Ractigen Therapeutics