｜Edited by the Content Team of Zhong肽 BiochemicalDecember 202425DayRactigen Therapeutics announced that its self-developed small nucleic acid innovative drug, RAG-17, has been included in the pilot project of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) under the "Patient-Centered Rare Disease Drug Development Pilot Program ('Care Program')".
"CARE Program (Patient-Centered Action for Rare Diseases Encouragement)" is dedicated to accelerating the research, development, and market entry of drugs for rare diseases by optimizing the review and approval processes, allowing more patients with rare diseases to benefit sooner. As a national pilot program, the "CARE Program" provides policy support addressing core challenges faced in the development of drugs for rare diseases, including early communication and expedited approval, empowering the entire process from drug development to market launch.According to the inclusion criteria of the CDE’s pilot project, the "Care Program" will select 1-2 representative and clinically valuable products for inclusion in the program across five application stages: A, B, C, D, and E. The successful selection of RAG-17 this time reflects the high recognition of its innovation and potential clinical value.Dr. Longcheng Li, founder and CEO of Ractigen, stated: "The successful inclusion of RAG-17 in the 'Care Program' is an affirmation of our team's relentless efforts in the field of rare disease drug development. This recognition brings us one step closer to our goal of providing a breakthrough treatment for ALS patients. We will fully leverage the 'Care Program.'“Provided resources and support to accelerate the development and market launch of RAG-17, bringing hope to more ALS patients and their families.”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 independently developed SCAD™ (Smart Chemical-Assisted Delivery) platform, can efficiently inhibit the expression of the SOD1 gene, 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.And in the IIT study,Intrathecal InjectionRAG-17 demonstrated good tolerability and safety at all dose levels, and the comprehensive safety assessment further supports the feasibility of its clinical development.RAG-17 was granted Orphan Drug Designation (ODD) by the U.S. FDA in 2023, andIn 2024, it successively obtained clinical trial approvals from the U.S. FDA and China CDE. Currently, Phase I clinical trials have been initiated in China, and the first subject has completed dosing, bringing new hope for ALS patients.About Ractigen
Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market, committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid pharmaceutical companies that simultaneously possess both intrahepatic and extrahepatic delivery technologies, having developed several internationally leading proprietary small nucleic acid drug delivery platform technologies, such as SCAD™ and LiCO™. 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 diseases, addressing undruggable targets and currently incurable conditions across various disease areas.Provide innovative treatment solutions for diseases.
