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The Foundation Fighting Blindness Clinical Research Institute Partnership to Accelerate Development of Gene Therapy for Stargardt Disease
10/02/2006

The Clinical Research Institute, the clinical trial support organization of the Foundation Fighting Blindness, announced today an agreement with Oxford BioMedica, a biopharmaceutical company in the United Kingdom, to collaborate on the production of the StarGen™ gene replacement therapy for the treatment of Stargardt disease and other related retinal degenerative diseases. A primary goal of the agreement is to initiate a Phase I clinical trial of StarGen by the end of 2008.

"StarGen holds promise for being a highly effective treatment for people affected by recessive Stargardt disease, many forms of cone-rod dystrophy, and other retinal degenerative diseases caused by variations in the ABCA4 gene," says Steve Rose, Chief Research Officer, FFB. "These diseases cause substantial vision loss, often at an early age, and there are virtually no treatments available for them. StarGen has the potential to change the lives of hundreds of thousands of people affected by these vision-robbing conditions."

The agreement between the Institute and Oxford BioMedica includes the establishment of a joint steering committee to guide StarGen development. The two companies have also agreed upon mutually beneficial financial arrangements to facilitate product development and future marketing efforts.

"This agreement accomplishes a key goal of FFB. We are accelerating the clinical trial process, which enables us to get a promising treatment out more quickly to the people who need it," says Bill Schmidt, Chief Executive Officer, FFB. "Furthermore, the terms of the agreement foster a collaborative relationship, which will help ensure the success of the treatment."

FFB-funded researchers identified the link between ABCA4 and Stargardt disease, and conducted the pivotal preclinical studies that demonstrated the potential safety and efficacy of gene therapy for Stargardt and related diseases.

Stargardt disease is most often caused by a harmful variation in a gene called ABCA4. As a result of the variation, a critical protein involved in retinal waste removal is not produced. The build-up of toxic waste products in the retina causes progressive loss of vision. Other related diseases, such as cone-rod dystrophy, are also caused by variations in the ABCA4 gene. The StarGen treatment involves replacement of the unhealthy ABCA4 gene with a normal gene, thereby serving as a promising treatment for any retinal disease caused by ABCA4.


Genetic Therapy Pipeline for Retinal Disease
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Genetic Therapy Pipeline for Retinal Disease

Nutritional Pipeline for Retinal Disease
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Nutritional Pipeline for Retinal Disease

Neuroprotective Pipeline for Retinal Disease
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Neuroprotective Pipeline for Retinal Disease

Cellular Therapy Pipeline for Retinal Disease
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Cellular Therapy Pipeline for Retinal Disease


 
 
The FFB Clinical Research Institute is a Support Organization of The Foundation Fighting Blindness