Sino-US firm Frontera Therapeutics, Inc. has announced that it has received approval from the US Food and Drug Administration (FDA) to conduct a Phase II study assessing its recombinant adeno-associated virus (rAAV) gene therapy, FT-003, in patients with diabetic macular edema (DME). This follows previous clearance for a Phase II study in neovascular age-related macular degeneration (nAMD) in the United States.
Preclinical Data and Therapeutic Potential of FT-003
Preclinical data indicates that FT-003 can efficiently transfect multiple layers of retinal cells upon injection into the fundus, leading these cells to express and secrete anti-VEGF proteins. This action inhibits endothelial cell division and proliferation, reduces vascular permeability, and offers comprehensive therapeutic functions. The gene therapy is anticipated to provide long-term effectiveness for patients with nAMD and DME through a single injection.
Clinical Studies in China and Positive Phase I Results
In China, clinical studies for FT-003 in both nAMD and DME have begun enrolling subjects. Encouraging results from Phase I trials have shown significant improvements in visual acuity and retinal structure for patients with nAMD and DME after intravitreal injection of FT-003. Moreover, the demand for anti-VEGF treatment decreased by more than 80%, significantly reducing the treatment burden for patients suffering from these conditions.-Fineline Info & Tech
