Blab Tech
Ractigen Secures FDA and China IND Approvals for First-In-Class saRNA Eye Therapy
Ractigen Therapeutics announced on 31 July 2026 that its small activating RNA (saRNA) candidate RAG‑1C has received Investigational New Drug (IND) clearance from both the U.S. Food and Drug Administration (FDA) and China’s Center for Drug Evaluation (CDE). The approvals open the door for global clinical testing of the world’s first saRNA therapy aimed at treating eye disease.
RAG‑1C tackles proliferative vitreoretinopathy (PVR), a serious complication that can arise after retinal detachment surgery or severe eye trauma. The therapy employs Ractigen’s LiCO™ (Lipid‑Conjugated Oligonucleotide) platform to deliver a highly specific saRNA that reactivates the endogenous p21 (CDKN1A) gene. By boosting p21 protein levels in retinal pigment epithelium and fibroblast cells, RAG‑1C induces cell‑cycle arrest, halting abnormal cell proliferation and myofibroblast transformation without inducing cellular toxicity. The drug is administered as a single intravitreal injection during eye surgery.
PVR remains a significant unmet medical need, affecting 5–10 % of rhegmatogenous retinal detachment cases and up to 40 % of severe ocular trauma cases. Current treatment options are limited to surgical intervention, which carries a high risk of failure and recurrent detachment. No FDA‑approved pharmacologic therapy exists for PVR, underscoring the potential impact of RAG‑1C for patients and clinicians.
The FDA IND approval follows the March 2025 clearance from China’s NMPA/CDE, making RAG‑1C the first saRNA ocular therapy to receive dual regulatory approval in both the United States and China. The FDA decision was based on preclinical data demonstrating ocular tolerability, minimal systemic exposure, and a favorable safety profile in GLP toxicology studies. The CDE clearance similarly reflected confidence in the drug’s safety and the robustness of the LiCO™ delivery system.
Ractigen plans to launch a Phase I clinical trial later in 2026. The study will enroll patients undergoing retinal detachment surgery who are at high risk of developing PVR. Primary objectives will be to assess safety, tolerability, pharmacokinetics, and preliminary efficacy of a single intraoperative intravitreal injection of RAG‑1C.
Dr. Long‑Cheng Li, founder and CEO of Ractigen, said the FDA clearance “marks a pivotal achievement for Ractigen and reinforces our leadership in RNA activation technology.” He added that the approval validates both the therapeutic potential of RAG‑1C and the LiCO™ platform, and that the company looks forward to initiating clinical studies to bring the therapy to patients worldwide.
Ractigen’s broader pipeline focuses on saRNA therapeutics for oncology, neurological disorders, and genetic diseases. The company’s LiCO™ platform is a non‑LNP, conjugated delivery technology that has been validated across multiple programs for tissue‑targeted delivery and ocular tolerability. The company’s strategy positions it to address other unmet medical needs where localized gene activation could provide therapeutic benefit.
In summary, Ractigen’s dual IND approvals establish a clear path for global clinical development of RAG‑1C. The company is preparing to start a Phase I trial in 2026, with the goal of addressing a critical gap in PVR treatment. Regulatory milestones, a robust delivery platform, and a clear unmet need set the stage for the next phase of evaluation.
RAG‑1C tackles proliferative vitreoretinopathy (PVR), a serious complication that can arise after retinal detachment surgery or severe eye trauma. The therapy employs Ractigen’s LiCO™ (Lipid‑Conjugated Oligonucleotide) platform to deliver a highly specific saRNA that reactivates the endogenous p21 (CDKN1A) gene. By boosting p21 protein levels in retinal pigment epithelium and fibroblast cells, RAG‑1C induces cell‑cycle arrest, halting abnormal cell proliferation and myofibroblast transformation without inducing cellular toxicity. The drug is administered as a single intravitreal injection during eye surgery.
PVR remains a significant unmet medical need, affecting 5–10 % of rhegmatogenous retinal detachment cases and up to 40 % of severe ocular trauma cases. Current treatment options are limited to surgical intervention, which carries a high risk of failure and recurrent detachment. No FDA‑approved pharmacologic therapy exists for PVR, underscoring the potential impact of RAG‑1C for patients and clinicians.
The FDA IND approval follows the March 2025 clearance from China’s NMPA/CDE, making RAG‑1C the first saRNA ocular therapy to receive dual regulatory approval in both the United States and China. The FDA decision was based on preclinical data demonstrating ocular tolerability, minimal systemic exposure, and a favorable safety profile in GLP toxicology studies. The CDE clearance similarly reflected confidence in the drug’s safety and the robustness of the LiCO™ delivery system.
Ractigen plans to launch a Phase I clinical trial later in 2026. The study will enroll patients undergoing retinal detachment surgery who are at high risk of developing PVR. Primary objectives will be to assess safety, tolerability, pharmacokinetics, and preliminary efficacy of a single intraoperative intravitreal injection of RAG‑1C.
Dr. Long‑Cheng Li, founder and CEO of Ractigen, said the FDA clearance “marks a pivotal achievement for Ractigen and reinforces our leadership in RNA activation technology.” He added that the approval validates both the therapeutic potential of RAG‑1C and the LiCO™ platform, and that the company looks forward to initiating clinical studies to bring the therapy to patients worldwide.
Ractigen’s broader pipeline focuses on saRNA therapeutics for oncology, neurological disorders, and genetic diseases. The company’s LiCO™ platform is a non‑LNP, conjugated delivery technology that has been validated across multiple programs for tissue‑targeted delivery and ocular tolerability. The company’s strategy positions it to address other unmet medical needs where localized gene activation could provide therapeutic benefit.
In summary, Ractigen’s dual IND approvals establish a clear path for global clinical development of RAG‑1C. The company is preparing to start a Phase I trial in 2026, with the goal of addressing a critical gap in PVR treatment. Regulatory milestones, a robust delivery platform, and a clear unmet need set the stage for the next phase of evaluation.
