AdPEDF is under development for the treatment of wet age-related macular degeneration (AMD). AMD is a progressive eye disease characterized by the growth of abnormal blood vessels in the center portion of the retina (the macula), resulting in loss of vision. GenVec believes that early intervention therapy with AdPEDF holds the promise to stabilize or improve vision in patients with AMD.
AdPEDF uses GenVec’s proprietary adenovector, a DNA carrier, to deliver the human Pigment Epithelium - Derived Factor (PEDF) gene, resulting in the local production of AdPEDF in the treated eye. PEDF is normally produced in the eye and serves two important functions. PEDF regulates normal blood vessel growth and protects the photoreceptors (the vision - sensing cells of the eye responsible for sight). AdPEDF’s key differentiation from other therapies is its potential to protect the photoreceptors damaged by fragile, leaky, abnormal blood vessels and inhibit blood vessel growth. In numerous animal studies, AdPEDF has been shown to rapidly elevate the intraocular AdPEDF protein levels in the eye, inhibit abnormal blood vessel growth, and cause abnormal blood vessels to regress while protecting the eye’s photoreceptors.
GenVec has completed the dose escalation portion of a Phase I, multi-center clinical trial in 28 patients with advanced wet AMD. Despite the advanced stage of disease in the patients studied, investigators observed positive changes in vision and retinal appearance at the higher dose cohorts in some patients. No dose limiting toxicities or drug related severe adverse events were observed. Additionally, GenVec has completed enrollment of 22 patients with less severe disease in this Phase I study. Very much like the first 28 patients, no dose limiting toxicities or drug related severe adverse events were observed. Investigators are continuing to collect data from this last cohort of 22 patients.
AMD is the leading cause of blindness in patients over the age of 50. Approximately 200,000 cases of AMD are diagnosed each year in the U.S. and more than 500,000 cases are diagnosed worldwide. Due to its potent multi-action antiangiogenic and neuroprotective activity, AdPEDF holds great promise to treat additional eye diseases, including diabetic retinopathy, diabetic macular edema, dry AMD, glaucoma, and retinitis pigmentosa. These diseases along with wet AMD will afflict more than 50% of the elderly population and represent multiple large commercial opportunities.
View previous clinical and pre-clinical data here
View product candidate summary here
