NANTONG and SUZHOU, China, Dec. 24,
2024 /PRNewswire/ -- Ractigen Therapeutics, a
clinical-stage pharmaceutical company dedicated to developing
RNA-based innovative therapies, today announced the successful
dosing of the first patient in the Phase I clinical trial of
RAG-17, an innovative siRNA therapy targeting Amyotrophic Lateral
Sclerosis (ALS) associated with superoxide dismutase 1 (SOD1)
gene mutations at Second Affiliated Hospital of Zhejiang University
School of Medicine.
The Phase I clinical study is a randomized, double-blind,
placebo-controlled trial designed to evaluate the
safety/tolerability, pharmacokinetics, pharmacodynamics, and
preliminary efficacy of RAG-17 in patients with SOD-ALS. The trial
is being conducted under the leadership of Dr. Yilong Wang at Beijing Tiantan Hospital of
Capital Medical University and Dr. Zhiying
Wu at the Second Affiliated Hospital of Zhejiang University
School of Medicine, respectively, with cooperation from Dr. Huifang
Shang at West China Hospital of Sichuan University.
"The first patient dosed in the RAG-17 trial marks a pivotal
milestone in our mission to combat ALS, one of the most devastating
neurodegenerative diseases," said Dr. Long-Cheng Li, Founder and CEO of Ractigen
Therapeutics. "This achievement underscores our unwavering
commitment to advancing RNA-based therapies that have the potential
to transform the lives of patients and families affected by rare
and severe conditions like ALS."
RAG-17 received Orphan Drug Designation (ODD) from the U.S. Food
and Drug Administration (FDA) in March
2023, followed by the FDA's clearance of its Investigational
New Drug (IND) application. In May
2024, the IND was approved by the Center for Drug Evaluation
(CDE) of China's National Medical
Products Administration (NMPA). Most recently, in November 2024, an Investigator-Initiated Trial
(IIT) of RAG-17 delivered promising clinical data, further
validating its potential as a transformative therapy. The promising
IIT findings have been presented at the 27th National Conference of
Neurology, Neuroscience 2024, and the 35th International Symposium
on ALS/MND, earning strong validation and interest from the global
scientific community.
About RAG-17
RAG-17 is a siRNA specifically designed to suppress the SOD1
gene in ALS patients with pathogenic mutations. Utilizing
Ractigen's proprietary SCAD™ delivery platform, RAG-17 conjugated
siRNA with an accessory oligonucleotide (ACO) for enhanced delivery
into the central nervous system (CNS). Preclinical studies,
including those using the hSOD1G93A mouse model, have
demonstrated the remarkable therapeutic efficacy of RAG-17 in
ameliorating motor function and prolonging survival. The IIT
results showed that intrathecally administered RAG-17 was
well-tolerated across all dose levels with only mild adverse events
and comprehensive safety evaluations confirming its favorable
profile.
About ALS
ALS, a severe neurodegenerative disease with no cure,
significantly reduces life expectancy, with most patients
succumbing to respiratory failure within 3-5 years of diagnosis.
Initial symptoms typically include muscle cramps, twitching, and
weakness. These symptoms progress to difficulties with movement and
speech, the need for assisted breathing, paralysis, and ultimately
death. Mutations in the SOD1 gene account for approximately 20% of
familial ALS and 5% of the sporadic ALS cases.
About Ractigen Therapeutics
A leader in small activating RNA (saRNA) drug development,
Ractigen Therapeutics is at the forefront of developing saRNA drugs
utilizing RNA activation (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.
View original
content:https://www.prnewswire.com/news-releases/ractigen-therapeutics-announces-first-patient-dosed-in-phase-i-clinical-trial-for-rag-17-in-sod1-als-302338866.html
SOURCE Ractigen Therapeutics
