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Stargardt Gene Therapy Will Begin Trials in USA and France

March 23, 2011 - British company, Oxford BioMedica, has received approval to begin a new trial of a gene therapy for Stargardt disease in the United States. The company plans to begin a small phase 1/2 trial of the therapy, known as StarGen^TM, in the spring of 2011. The trial will enrol a total of 28 people with Stargardt disease in the United States and France. It will test three different dosage levels of the therapy for its safety and biological activity, and for the participant’s ability to tolerate the treatment. In the United States, the study will be led by Dr. Peter Francis at the Oregon Health and Science University, in Portland, Oregon.

Autosomal recessive Stargardt disease is caused by one of over 500 possible mutations in the ABCA4 gene. These genetic mutations cause the photoreceptors of the eye to slowly break down causing a gradual loss of vision. Most people with Stargardt disease are diagnosed in their teens, and slowly loss vision throughout life, beginning with the central part of their sight (visual field).

StarGen^TM therapy will use a specially engineered, harmless virus to insert healthy new ABCA4 genes into the cells of the retina, substituting these for the damaged genes. The goal is to restore the gene's missing function; if done early enough this should prevent blindness.  ABCA4 is a large gene, and gene therapy is technically difficult, however animal studies of this treatment suggest that a single application of StarGen^TM to the retina may be enough to provide long-term, or potentially permanent, correction. 

This initial trial is only for Stargardt disease however the ABCA4 gene may also play a role in other conditions. Current estimates suggest that 30-60% of cone-rod dystrophies may be due to this gene, and some evidence suggests it may also play a role in dry AMD.

Other research groups are also working on gene therapies for the ABCA4 gene including a group of investigators led by Dr. Robert Molday at the University of British Columbia. This group is funded by a grant from the Foundation Fighting Blindness and the Canadian Institutes of Health Research.

Oxford Biomedica is also testing gene therapies for other retinal degenerative diseases, including a gene therapy for wet AMD that is now in the early stages of human testing in  the United States, and an early trial of  gene therapy for Usher syndrome type 1B, which is expected to begin later this year.