Ractigen Therapeutics Secures U.S. FDA IND Clearance for First-in-Class saRNA Therapy RAG-1C Targeting Proliferative Vitreoretinopathy
核心洞察
Ractigen Therapeutics received U.S. FDA IND clearance for RAG-1C, a first-in-class small activating RNA (saRNA) therapy for proliferative vitreoretinopathy (搜索) (PVR), following prior clearance from China's CDE in March 2025.
RAG-1C utilizes the proprietary LiCO™ platform to activate the p21 gene, inducing cell cycle arrest to inhibit cellular proliferation and myofibroblast transformation without cellular toxicity.
PVR affects 5–10% of all rhegmatogenous retinal detachment cases and up to 40% of severe ocular trauma cases, with no FDA-approved pharmacological therapies currently available.
Ractigen Therapeutics, a clinical-stage biopharmaceutical company pioneering small activating RNA (saRNA) therapeutics, announced on July 31, 2026, that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for RAG-1C. This first-in-class saRNA drug candidate is designed for the prevention and treatment of proliferative vitreoretinopathy (搜索) (PVR), a severe, sight-threatening complication with no currently approved pharmacological therapies.
The FDA clearance follows the IND approval received from China's National Medical Products Administration (NMPA/CDE) in March 2025, positioning RAG-1C as the first saRNA therapy in ocular diseases to enter global clinical development in both the United States and China.
The Unmet Need in PVR
PVR is the leading cause of surgical failure in rhegmatogenous retinal detachment (RRD) repair, affecting 5–10% of all RRD cases and up to 40% of severe ocular trauma cases. The condition is driven by the abnormal proliferation and migration of retinal pigment epithelium (RPE) cells and fibroblasts, leading to excessive cellular proliferation and membrane formation on the retinal surface. This ultimately results in tractional retinal detachment and permanent vision loss. Currently, surgical intervention remains the only option for patients, yet it often suffers from high rates of failure and recurrent retinal detachment.
Mechanism of Action and the LiCO™ Platform
RAG-1C employs Ractigen's proprietary LiCO™ (Lipid-Conjugated Oligonucleotide) platform to deliver a highly specific saRNA that reactivates the endogenous p21 (CDKN1A) (搜索) gene. By upregulating p21 protein expression in target ocular cells, RAG-1C induces cell cycle arrest, effectively inhibiting cellular proliferation and myofibroblast transformation without cellular toxicity. The LiCO™ platform is a non-LNP, conjugated delivery technology optimized for tissue-targeted delivery and has been extensively validated across multiple clinical and preclinical programs, demonstrating exceptional ocular tolerability and minimal systemic exposure in extensive GLP toxicology studies.
Administered as an intravitreal injection (IVT), RAG-1C offers the potential for long-lasting anti-fibrotic protection through a single intraoperative administration, a concept the company describes as "One-and-Done."
Leadership Perspective
"Securing FDA IND clearance for RAG-1C marks a pivotal achievement for Ractigen and reinforces our leadership in RNA activation technology," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "Following our China CDE clearance last year, this global approval validates the safety and therapeutic potential of both RAG-1C and our proprietary LiCO™ platform. PVR represents a critical unmet clinical need with zero approved drug options. We look forward to initiating our clinical studies to bring this transformative therapy to patients worldwide."
Upcoming Clinical Development
The upcoming Phase I clinical trial will evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-1C in patients undergoing surgery for retinal detachment who are at high risk of developing PVR. The dual IND clearances from both U.S. and Chinese regulatory authorities pave the way for a global clinical development program for what would be the world's first saRNA therapy for ocular diseases.
Ractigen Therapeutics is advancing a robust pipeline addressing unmet medical needs across oncology, neurological diseases, and genetic disorders, leveraging additional proprietary delivery platforms including SCAD™ and GLORY™ alongside LiCO™.