｜Edited by the Content Team of Zhong肽 BiochemicalDecember 202424Day，Ractigen Therapeutics Announces First Dosing of RAG-17 in Phase I Clinical Trial for ALS Caused by SOD1 Gene Mutation at the Second Affiliated Hospital of Zhejiang University School of MedicineS treatment field has achieved a phased breakthrough, bringing new hope for solving the major problem of ALS.
This study is a randomized, double-blind, placebo-controlled Phase I clinical trial designed to evaluate the safety/tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in ALS patients carrying the SOD1 gene mutation. The study is led by Professor Wang Yilong, Chief Scientist of the Neurology Center at Beijing Tiantan Hospital, and Professor Wu Zhiying, Director of the Rare Disease Diagnosis and Treatment Center at the Second Affiliated Hospital of Zhejiang University School of Medicine, with the collaboration of Professor Shang Huifang, Deputy Director of the Department of Neurology at West China Hospital, Sichuan University.
Dr. Longcheng Li, Founder/CEO of Ractigen, stated: “The completion of dosing for the first subject in the RAG-17 trial marks an important step forward in our efforts to tackle amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease. This achievement highlights our relentless commitment to advancing RNA therapies, which hold the potential to bring life-changing hope to patients suffering from severe rare conditions like ALS and their families.”
RAG-17 was granted Orphan Drug Designation (ODD) by the U.S. FDA in March 2023 and subsequently received clinical trial approvals from both the FDA and China's Center for Drug Evaluation (CDE) of the National Medical Products Administration in 2024. In November 2024, RAG-17 demonstrated positive clinical outcomes in an Investigator-Initiated Trial (IIT), further validating its potential as a revolutionary therapy. These research findings were presented at the 27th Chinese Neurology Congress, Neuroscience 2024, and the 35th International ALS/MND Symposium, garnering significant attention and high praise.。RAG-17 is an innovative double-stranded small interfering RNA (siRNA) drug independently developed by Ractigen Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS) carrying SOD1 gene mutations. The drug, based on Ractigen's proprietary SCADTM (Smart Chemical-Assisted Delivery) platform, can efficiently inhibit SOD1 gene expression, thereby reducing the production of toxic proteins and protecting neuronal function.
In preclinical efficacy studies, RAG-17 significantly delayed disease onset, extended the survival of model animals, and markedly improved their motor function. Additionally, in the IIT study, intrathecal injection of RAG-17 demonstrated good tolerability and safety across all dose levels. The comprehensive safety evaluation further supports its feasibility for clinical development. RAG-17 received Orphan Drug Designation (ODD) from the U.S. FDA in 2023 and has successively obtained clinical trial approvals from the U.S. FDA and China CDE in 2024. Phase I clinical trials have now been initiated in China, bringing new hope for ALS patients.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 patients typically surviving 3-5 years after onset. ALS can be divided into sporadic and familial forms, with familial ALS often caused by mutations in various genes. SOD1 gene mutation is one of the most common pathogenic factors, accounting for approximately 20% of familial ALS and 5% of sporadic ALS cases. In China, SOD1 is the most common mutated gene causing ALS.Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market, dedicated to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid drug companies that simultaneously possess both intrahepatic and extrahepatic delivery technologies, having developed multiple proprietary delivery platform technologies with international leading standards, such as SCADTM and LiCOTM. Based on RNA activation technology and its self-developed Smart-TTC saRNA drug development platform, the company has established a highly differentiated small nucleic acid drug pipeline, with indications covering neurodegenerative diseases, neuromuscular diseases, cancer, metabolic and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.
