May 16, 2024
eMedClub News
May 15,The official website of the Center for Drug Evaluation (CDE) shows that Ractigen's RAG-17 Injection has received approval for a new drug clinical trial (IND).）The application has received implied permission and is intended for the treatment of adults with superoxide dismutase 1 (SOD1) Mutation-induced Amyotrophic Lateral Sclerosis (ALS)。 ▲ Source of the image: CDE official website
RAG-17 is a targeted and inhibitorySOD1Double-stranded small interfering RNA (siRNA) for gene expression，Multiple preclinical efficacy studies have shown that RAG-17 exhibits superior efficacy compared to benchmark drugs in ALS disease models (e.g., the hSOD1G93A mouse model).Last year,RAG-17Granted FDA Orphan Drug Designation (ODD)Recognition, used for the treatment of Amyotrophic Lateral Sclerosis (ALS).ALS is a chronic progressive neurodegenerative disease characterized by the prominent involvement of upper and lower motor neurons, primarily manifesting as muscle weakness, muscle atrophy, bulbar paralysis, and pyramidal tract signs. ALS is an incurable disease with a typical survival period of 3-5 years. ALS can be divided into sporadic and familial forms. Familial ALS can be caused by mutations in various genes, including superoxide dismutase 1 (SOD1) is one of the common genes. In ChinaSOD1It is the most common mutation gene that causes ALS.Approximately 20% of familial ALS and 5% of sporadic ALS areSOD1Caused by gene mutations。RAG-17Applied Ractigen's independently developed Smart Chemical-Assisted Delivery (SCAD).）Platform technology that enables efficient and long-lasting delivery in the central nervous system (CNS) by conjugating double-stranded RNA with auxiliary oligonucleotides (ACO).▲ SCADIntroduction to Delivery Systems
Data shows,SCAD'sMain features include：- Direct CNS Delivery: Addressing unmet needs for neurological disorders requiring direct central nervous system intervention.
- Wide Biological Distribution: Achieving extensive tissue distribution and therapeutic effects in central nervous system tissues.
- Sustained Delivery Activity:Achieve at least 3 months of sustained target gene silencing。
- High Safety:Its safety has been validated in GLP toxicology studies and in patients.
In April this year,Dr. Longcheng Li, Founder/CEO of Ractigen, onceRepresentation，“Mr. Cai Lei is not only aAn outstanding entrepreneur, and a brave fighter against ALS. For many years, he has been actively fighting against the disease, speaking up for patients with ALS, and dedicating himself to advancing the research and development of ALS drugs.BingAdhering to the principles of innovation and clinical demand-oriented project initiation, we leverage our globally leading expertise in the central nervous system SCAD™ Delivery Technology, Ractigen Therapeutics has established multiple new drug pipelines for the treatment of ALSLine.RemarkableExcitingly, our first new drug for ALS, RAG-17, has been developed after more than three years of dedicated research.HairHas entered the clinicalBed validation, preliminary results show positive efficacy and good safety.RAG-17 received full support and assistance from Mr. Lei Cai and his team during the development process., including the completion of a key preclinical pharmacodynamics study for our project and the acquisition of conclusive pharmacodynamics data.In the future, both parties will continue to leverage their respective resource advantages, deepen and expand the cooperation model, empower the development and transformation of ALS drugs, and strive to bring the drugs to patients as soon as possible, offering hope for survival to the patients.”Mr. Cai Lei said, "Over the past four years, we have been working on the ALS rare disease research data platform in China, patientPatientBreakthroughs have been made in areas such as demand aggregation, genomics analysis, proteomics, metabolomics, pathology database construction, iPSC, and rapid drug screening using organoids. Collaborations with many pharmaceutical companies have also accelerated the development of drugs for ALS.Ractigen RAG-17 DrugThe efficacy of the drug is globally leading, and I highly value this drug and the hope it brings.”Recommended Thematic Forums
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Reference Source:CDE OfficialNetwork, Public InformationStatement and Copyright Notice
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