Phase I/II Study of Retinal Gene Therapy for X-linked Retinitis Pigmentosa
What is the study about and what is it trying to achieve?
A Phase I/II clinical trial of gene therapy for X-linked Retinitis Pigmentosa run by Biogen in collaboration with Professor Robert MacLaren (Oxford team) and previously Professor Paulo Stanga (Manchester team).
X-linked Retinitis Pigmentosa (XLRP) is an inherited genetic condition that results in progressive retinal degeneration, which eventually leads to blindness. The disease is caused by a mutations in the RPGR gene located on the X-chromosome. As men have one X chromosome and women have two x chromosomes, the disease affects men and women differently.
This study has now closed to recruitment.