Investigators Move Closer to Gene Therapy Trial for Choroideremia

Researchers are reporting that a clinical trial for gene therapy to treat choroideremia may be two years away thanks to successes in preclinical studies of gene therapy for choroideremia and human studies of gene therapy to treat Leber congenital amaurosis (LCA). 

Jean Bennett, M.D., Ph.D., of the University of Pennsylvania, is developing a gene therapy for choroideremia using an adeno-associated virus, a delivery technology that is similar to the one she used in a landmark clinical trial of LCA gene therapy that restored vision to children and young adults. Dr. Bennett says that after additional preclinical work, she could potentially begin a gene therapy clinical trial for choroidermia in 2012.

Miguel Seabra, Ph.D., a Foundation-funded researcher from Imperial College London, and Robert MacLaren from the University of Oxford, are part of a collaborative team that is also working toward a clinical trial of gene therapy for choroidermia.