Applied Genetic Reports Positive Preclinical Data for Eye Disorder Treatment
Applied Genetic Technologies Corporation (AGTC) announced the results for its clinical trial related to X-Linked retinitis pigmentosa. The data provided the basis for determining the starting dose and the vector for its current Phase 1/2 clinical trial. The results demonstrated that the company's proprietary AAV vector and engineered RPGR constructs were well tolerated. It also had a positive impact on markers of disease in a canine model of XLRP.
Full length RPGR protein's DNA sequence may contain repetitive sequences which may cause instability during vector