Ractigen Therapeutics Announces CDE Approval of IND for RAG-1C, China's First saRNA Drug to Enter Clinical Trials for Proliferative Vitreoretinopathy
Ractigen
Innovative Drug R&D Developer
Previously, Ractigen's first saRNA drug, RAG-01, received IND approval from the U.S. FDA.Positive preliminary efficacy and safety data were obtained in the Phase I clinical trial conducted in Australia.RAG-1C is the second saRNA drug developed by Ractigen Therapeutics to enter clinical trials.
RAG-1C inhibits the abnormal proliferation and migration of retinal pigment epithelial (RPE) cells by upregulating p21 gene expression, thereby preventing the progression of PVR. PVR is a severe complication after rhegmatogenous retinal detachment (RRD) repair surgery, characterized by the formation of subretinal and/or epiretinal fibrotic membranes, often leading to retinal re-detachment and potentially resulting in significant vision loss or even blindness.Currently, there are no approved drugs specifically for the treatment of PVR, and vitreoretinal surgery remains the primary treatment method, with significant unmet clinical needs.
This Phase I clinical trial is a multi-center, randomized, double-blind, dose-escalation first-in-human study designed to evaluate the safety/tolerability, pharmacokinetics, and preliminary efficacy of RAG-1C in preventing proliferative vitreoretinopathy after vitrectomy.
Dr. Longcheng Li, founder and CEO of Ractigen, stated:"RAG-1C is a globally pioneering RNA therapy that holds the potential to offer a breakthrough treatment option for PVR patients. The approval of the CDE clinical trial permit has made RAG-1C the first saRNA drug in China to receive clinical approval, showcasing the cutting-edge nature of RNA activation technology and demonstrating the full recognition by Chinese regulatory authorities of this innovative mechanism. Given the complex pathological mechanisms of PVR and the long-standing lack of effective drug treatments, we believe RAG-1C, through its precise gene regulation mechanism, can provide patients with a new therapeutic choice, filling the gap in the PVR treatment field."
AboutRAG-1C
RAG-1C is an saRNA drug that upregulates the expression of the p21 gene, a negative regulator of the cell cycle, through the RNAa mechanism, thereby inhibiting the proliferation and migration of retinal pigment epithelial cells. In preclinical studies, Ractigen Therapeutics' self-developed LiCO™ small nucleic acid delivery system was used for intravitreal injection.RAG-1C demonstrated an extremely significant effect in inhibiting disease progression in the rabbit PVR model, strongly proving its therapeutic potential.
About RVR
Proliferative Vitreoretinopathy (PVR) is a severe fibrotic complication of rhegmatogenous retinal detachment surgery, with an incidence rate of 5-10%. It is the primary cause of retinal re-detachment and surgical failure, accounting for 75% of re-detachment cases. PVR is characterized by the abnormal migration and proliferation of RPE cells into the vitreous cavity, forming contractile fibrocellular membranes that lead to retinal re-detachment and transform rhegmatogenous retinal detachment (RRD) into tractional retinal detachment (RD), potentially resulting in irreversible vision loss. Despite decades of research, there are currently no FDA-approved drugs for PVR treatment, and surgery remains the only therapeutic option.
About RNAa
RNA Activation is a platform technology pioneered internationally by Dr. Li Longcheng, the founder of Ractigen Therapeutics, and his team, which has already been clinically validated. This technology uses double-stranded RNA targeting the promoter region of genes to activate gene expression, restoring the levels of therapeutic proteins. RNA activation is a highly scarce platform technology in the pharmaceutical field, with broad application prospects in drug development, including genetic diseases, chronic diseases, metabolic diseases, cardiovascular and cerebrovascular diseases, and cancer.
AboutRactigen
Ractigen Therapeutics is a clinical-stage biopharmaceutical company 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 capabilities, having developed several internationally leading small nucleic acid drug delivery platform technologies with independent intellectual property rights, including SCAD™, LiCO™, and GLORY™. 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 and neuromuscular diseases, cancer, metabolic and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.