SUZHOU, China, May 21, 2024
/PRNewswire/ -- Ractigen Therapeutics, a pioneering developer of
small activating RNA (saRNA) therapeutics, proudly announces that
its flagship program, RAG-01, has been granted Fast Track
Designation (FTD) by the U.S. Food and Drug Administration (FDA).
This notable achievement marks a significant milestone in the
advancement of saRNA technology and underscores Ractigen's
commitment to addressing critical unmet medical needs. The
milestone establishes RAG-01 as the first saRNA drug worldwide to
achieve FTD.
RAG-01 is currently undergoing a Phase I clinical trial in
Australia for the treatment of
non-muscle invasive bladder cancer (NMIBC). The trial, initiated in
December 2023, has successfully
enrolled and dosed three patients, demonstrating the program's
progress in clinical development.
FDA's recent approval of the Investigational New Drug (IND)
application for RAG-01 further validates the therapeutic potential
of this innovative saRNA therapy. This regulatory milestone not
only paves the way for the expansion of clinical trials in
the United States but also
highlights the FDA's recognition of RAG-01's promise in addressing
the urgent medical needs of NMIBC patients.
Fast Track Designation is granted to investigational drugs
intended for the treatment of serious conditions with unmet medical
needs, facilitating their expedited development and review process.
With FTD, Ractigen gains enhanced opportunities for collaboration
with the FDA, enabling closer communication and expedited guidance
throughout the development and regulatory review process.
Dr. Long-Cheng Li, Founder and
CEO of Ractigen Therapeutics, expressed his enthusiasm about the
FDA's decision: "We are thrilled to receive Fast Track Designation
for RAG-01, marking a significant milestone not only for our
program but also for the saRNA field as a whole. This designation
underscores the urgency and importance of advancing innovative
therapies like RAG-01 to address critical medical needs. We
remain dedicated to accelerating the development of innovative
saRNA therapies to address a wide range of diseases, including
cancer, genetic disorders, and chronic conditions. Through
strategic collaborations and pioneering research efforts, the
company aims to deliver transformative treatments that improve
patient outcomes and quality of life."
About RAG-01: RAG-01 is a pioneering saRNA candidate
engineered to target and activate the tumor suppressor gene p21 via
the mechanism of RNAa. Traditionally considered "undruggable," p21
presents a unique opportunity for saRNA-based targeted activation.
The drug, delivered through intravesical instillation using
Ractigen's proprietary LiCO™ delivery technology, has shown
significant tumor suppression in mouse orthotopic bladder cancer
models. Currently, the Phase I clinical trial of RAG-01 in
Australia has successfully
enrolled and dosed the first three patients. Its development marks
a significant stride in RNAa based therapies, addressing the unmet
needs of NMIBC patients.
About NMIBC: NMIBC represents 50-80% of all bladder
cancer cases. Despite standard treatments like transurethral
resection of bladder tumor (TURBT) followed by intravesical BCG or
chemotherapy, recurrence rates remain high, estimated at 50-70%
within the first five years. RAG-01's development is a significant
step towards addressing this substantial unmet need in bladder
cancer therapy.
About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically
validated platform technology. It employs saRNA to target gene
regulatory domains, activating gene expression and restoring
therapeutic protein levels. This technology has vast potential for
developing therapeutic drugs across various diseases, especially
where traditional methods fall short, including cancer, genetic
disorders, chronic diseases, and metabolic and cerebrovascular
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