Ractigen Therapeutics Announces U.S. FDA IND Clearance for First-in-Class saRNA Candidate RAG-1C to Treat Proliferative Vitreoretinopathy

• 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

NANTONG, China, July 31, 2026 /PRNewswire/ -- Ractigen Therapeutics, a pioneer and global leader in small activating RNA (saRNA) therapeutics, today 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.

About RAG-1C and the LiCO™ Platform

RAG-1C is a first-in-class saRNA therapeutic candidate targeting the human p21 gene. Delivered using Ractigen's proprietary LiCO™ platform, RAG-1C is designed to achieve sustained, localized gene activation in ocular tissues following intravitreal injection. 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.

About Proliferative Vitreoretinopathy (PVR)

Proliferative Vitreoretinopathy (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. Characterized by excessive cellular proliferation and membrane formation on the retinal surface, PVR leads to tractional retinal detachment and permanent vision loss. There are currently no FDA-approved drugs for PVR.

About Ractigen Therapeutics

Ractigen Therapeutics is a clinical-stage biopharmaceutical company innovating next-generation RNA therapeutics, with a primary focus on small activating RNAs (saRNAs) developed through its clinically validated RNA activation (RNAa) technology. Leveraging proprietary delivery platforms such as SCAD™, LiCO™, and GLORY™, Ractigen is advancing a robust pipeline addressing unmet medical needs in oncology, neurological diseases, and genetic disorders. Its versatile technologies also enable the rapid development of RNA-based solutions, including siRNAs, where applicable, to target life-threatening, fast-progressing conditions such as those in the CNS. Committed to scientific excellence and patient-centered innovation, Ractigen strives to transform healthcare through the power of RNA therapeutics. For more information, visit www.ractigen.com.

SOURCE Ractigen Therapeutics