APLS - Applied Genetic Reports Positive Data And Other News: The Good Bad And Ugly Of Biopharma
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