NNRI
Partnership to Accelerate Development of Gene Therapy for Stargardt
Disease
10/02/2006
The National
Neurovision Research Institute (NNRI), 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 NNRI 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. |
