NightstaRx and The University of Alberta announce the start of the first Canadian gene therapy study to treat Choroideremia

NightstaRx Ltd (“Nightstar”), the biopharmaceutical company specialising in bringing therapies for retinal dystrophies to patients, announces that the University of Alberta, has begun enrolling and dosing subjects in a Phase II clinical trial of the Company’s gene therapy for the treatment of choroideremia (CHM). This gene therapy approach uses a viral vector known as adeno-associated virus (AAV) to deliver a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) into cells of the eye.

The trial, which is sponsored by the University of Alberta, is an open label study involving a total of 6 male patients, who will each receive a single dose of AAV2-REP1 via a sub retinal injection.

Choroideremia is an inherited X-linked recessive disease which inevitably causes blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 and affects approximately 1 in 50,000 people. The first symptom of the condition is usually an impairment of night vision which often occurs in early childhood. This is followed by progressive narrowing of the field of vision, as well as a decrease in the ability to see details, culminating in blindness, most commonly in late adulthood. No effective treatment currently exists.

David Fellows, CEO of Nightstar said:
“Gene therapy treats genetic diseases at the molecular level by correcting what is wrong with defective genes. We are broadening our pipeline of products in development and are leading the way in the development of an effective gene therapy treatment for choroideremia. This new study, sponsored by the University of Alberta, is another step forward in the development of AAV2-REP1. We have been granted Orphan Drug Designation for the product in the United States and Europe and the data to date has shown very promising results.”

Ian MacDonald, Professor of Ophthalmology and Visual Sciences, University of Alberta commented:
“We are very excited to be working with Nightstar and to have treated our first choroideremia patient. 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. The next challenge will be making this therapy available to all individuals with the condition as soon as we possibly can.”

About Choroideremia

Choroideremia is an inherited disorder that leads to progressive loss of vision due to degeneration of the choroid and retina which is caused by a lack of Rab Escort Protein-1 (REP-1) and occurs almost exclusively in males. The first symptoms occur in childhood, with night blindness being the most common first symptom. As the disease progresses, there is loss of peripheral vision or ‘tunnel vision’, and later a loss of central vision. Progression of the disease continues throughout the individual’s life, although both the rate and the degree of visual loss can vary, even within the same family. There is currently no treatment or cure for this disease.