• RAG-1C achieves dual IND clearances from both U.S. FDA and China CDE, paving the way for global clinical development of the world's first saRNA therapy for ocular diseases
• RAG-1C utilizes Ractigen's clinically validated LiCO™ platform to target p21 gene activation for long-lasting anti-fibrotic efficacy

Ractigen Therapeutics, a pioneer and global leader in small activating RNA (saRNA) therapeutics, announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for RAG-1C, a first-in-class saRNA drug candidate designed for the prevention and treatment of Proliferative Vitreoretinopathy (PVR).

This milestone follows the IND approval received from China's National Medical Products Administration (NMPA/CDE) in March 2025, making RAG-1C the first saRNA therapy in ocular diseases to enter global clinical development in both the United States and China.

PVR is a severe, sight-threatening complication that occurs following rhegmatogenous retinal detachment (RRD) or ocular trauma, driven by the abnormal proliferation and migration of retinal pigment epithelium (RPE) cells and fibroblasts. Currently, there are no FDA-approved pharmacological therapies for PVR, leaving surgical intervention as the only option, which often suffers from high rates of failure and recurrent retinal detachment.

RAG-1C utilizes Ractigen's proprietary LiCO™ (Lipid-Conjugated Oligonucleotide) platform to deliver a highly specific saRNA that reactivates the endogenous p21 (CDKN1A) 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. Administered as an intravitreal injection (IVT), RAG-1C offers the potential for long-lasting anti-fibrotic protection through a single intraoperative administration ("One-and-Done").

"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, 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."

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.