Ractigen wins FDA IND clearance for saRNA candidate

Ractigen Therapeutics has secured clearance from the U.S. Food and Drug Administration for its Investigational New Drug application for RAG-1C, marking a major regulatory milestone for a first-in-class small activating RNA candidate [1.1]. The therapy is specifically designed for the prevention and treatment of proliferative vitreoretinopathy, a severe and sight-threatening condition.

The decision by American regulators follows a similar Investigational New Drug approval granted by China's National Medical Products Administration in March 2025. These dual clearances make RAG-1C the first small activating RNA therapy targeting ocular diseases to advance into global clinical development across both the United States and China.

Proliferative vitreoretinopathy is a frequent and severe complication that can arise following rhegmatogenous retinal detachment repair or acute ocular trauma. Driven by the abnormal proliferation of retinal pigment epithelium cells and fibroblasts, the condition often leads to tractional retinal detachment and permanent vision loss.

Currently, medical science lacks any FDA-approved pharmacological treatments for the condition, leaving patients dependent on high-risk surgical interventions. These surgical procedures frequently suffer from high failure rates and subsequent recurrent retinal detachments, underscoring an acute clinical need for effective drug alternatives.

RAG-1C leverages Ractigen's proprietary Lipid-Conjugated Oligonucleotide delivery platform to transport specialized small activating RNA directly into target ocular tissues. By reactivating the endogenous p21 gene, the treatment induces cell cycle arrest in problematic cells, effectively blocking cellular proliferation and myofibroblast transformation without cellular toxicity.

Delivered via an intravitreal injection during surgery, the candidate is engineered to provide long-lasting anti-fibrotic protection through a single intraoperative administration. Company executives noted that the global regulatory progress validates both the underlying technology platform and the therapeutic strategy for addressing severe unmet medical needs.

With regulatory clearance secured, Ractigen prepares to launch an initial Phase I clinical trial. The upcoming study will evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-1C in patients undergoing retinal detachment surgery who face a high risk of developing proliferative vitreoretinopathy.