GenAns' GA001 gene therapy cleared by FDA for Phase II clinical trial

Recently, GenAns Biotech has reached a significant R&D milestone: its self-developed gene therapy drug, GA001 injection for the treatment of retinitis pigmentosa (RP), has demonstrated a favorable safety profile in prior clinical studies. Following evaluation by the U.S. Food and Drug Administration (FDA), the drug has now officially received clinical trial approval, authorizing the initiation of a Phase II clinical study to evaluate its efficacy and safety.

About GA001 Injection

GA001 injection is a novel gene therapy drug independently developed by GenAns Biotech for patients with blindness. Its core technology is a novel, highly sensitive photosensitive protein discovered by the GenAns research team. Utilizing an adeno-associated viral (AAV) vector, the drug delivers the gene encoding this novel photosensitive protein to retinal ganglion cells, restoring their light-sensing function and ultimately helping patients regain vision.

Updates on Previous IIT Progress

The Investigator-Initiated Trial (IIT) of GA001 injection conducted at Beijing Tiantan Hospital completed the enrollment and dosing of its first subject in January 2024. To date, the study has achieved interim results: all patients have experienced varying degrees of visual improvement, with some able to identify letters and patterns, significantly enhancing their ability for independent daily living. No drug-related serious adverse events have been observed, preliminarily confirming the favorable safety profile and significant therapeutic potential of GA001.