Alberta, Canada—Gene therapy to treat choroideremia is one step closer to reality thanks to a new study.
“Choroideremia is a devastating condition for individuals and families, but we believe our new gene therapy will arrest any further deterioration of vision and will provide long lasting benefit,” said Ian MacDonald, MD, CM, principal investigator of the new phase II clinical trial, which is sponsored by the Faculty of Medicine and Dentistry of the University of Alberta, Canada
Dr. MacDonald expressed his excitement at being involved in the project and said he had already treated his first patient.
Additionally, the biopharmaceutical company NightstaRx Ltd (Nightstar) has developed a highly promising new gene therapy product for this rare disorder.
The product has been granted orphan drug designation in both the United States and Europe.
David Fellows, chief executive officer of Nightstar, explained that the company— which specialises in therapies for retinal dystrophies—is “broadening (its) pipeline of products in development and . . . leading the way in the development of an effective gene therapy treatment for choroideremia.”
Gene therapy for choroideremia
Because the genetics of choroideremia are well understood, it has long been considered a suitable target for gene therapy, in particular because almost all known cases involve functionally null mutations to the relevant gene.