Cellgate begins AMD study
CGC-11047 is a novel polyamine analog that targets the hyper-proliferating blood vessel growth, known as choroidal neovascularization, associated with age-related macular degeneration (AMD). The phase I trial is
Ultragenyx Pharmaceutical has received accelerated approval from the US Food and Drug Administration (FDA) for its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr), to treat glycogen storage disease type Ia (GSDIa).