Sunday, May 10, 2009

Foundation Fighting Blindness’ National Neurovision Research Institute Heralds Collaboration for Gene Therapy Advancements

Foundation Fighting Blindness’ National Neurovision Research Institute Heralds Collaboration for Gene Therapy Advancements

OWINGS MILLS, Md.--(BUSINESS WIRE)--The National Neurovision Research Institute (NNRI), the Foundation Fighting Blindness’ clinical trial support organization, announced today that one of its key partners, biopharmaceutical company Oxford BioMedica, has established a collaboration with sanofi-aventis, a major international pharmaceutical company, to develop and commercialize gene therapy treatments for vision-robbing retinal degenerative diseases that affect tens of millions of people around the world.

The collaboration will significantly bolster the development of the following gene therapy products: StarGen™ for the treatment of Stargardt disease, a form of early-onset macular degeneration; UshStat™ for the treatment of Usher syndrome, the leading cause of deaf-blindness; RetinoStat® for the treatment of age-related macular degeneration, the leading cause vision loss in people 55 and older in developed countries; and EncorStat™ for corneal graft rejection.

The development of treatments for these and other retinal degenerative diseases is a key goal of the National Neurovision Research Institute and is the basis for its partnership with Oxford BioMedica. In 2006, Paul and Diane Manning, with the National Neurovision Research Institute, established a partnership with Oxford BioMedica to advance gene therapies for these and other related retinal degenerative diseases.

“The investment from sanofi-aventis is a wonderful boost for the development of Oxford BioMedica’s gene therapy products and will greatly enhance our ability to move these emerging treatments into and through the clinical trial process,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “This collaboration affirms the great potential for gene therapy to treat and cure a number of retinal degenerative diseases including Stargardt disease and Usher syndrome that, as rare diseases, often do not receive the attention or investment necessary to bring about promising treatments.”

“An important goal of the Foundation, through NNRI, is to attract investment from large pharmaceutical companies for the development and production of treatments for inherited retinal disease,” says Morton Goldberg, M.D., chairman of the board of NNRI. “The NNRI-Oxford partnership is an excellent model of how NNRI collaborations can accelerate the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like inherited retinal degenerations to the market and out to the people who need them.”

Based on the agreement, Oxford BioMedica will receive approximately €43 million ($56 million) from sanofi-aventis over a three-year period. Oxford BioMedica is eligible to receive additional fees if development efforts are successful.

The treatments will utilize Oxford BioMedica’s LentiVector® gene delivery technology to deliver healthy vision-saving genes to the retina.

About Foundation Fighting Blindness

The Foundation Fighting Blindness is the largest source of non-governmental funding for retinal degenerative disease research in the world. The urgent mission of the Foundation Fighting Blindness is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases. The Foundation has invested over $140 million to provide seed money for scientific research of diseases of the retina, which cause blindness. Further information is available at www.FightBlindness.org.

About National Neurovision Research Institute (NNRI)

NNRI is a recently-established non-profit support organization of the Foundation Fighting Blindness (FFB), the leading non-government funding source for inherited orphan retinal degeneration research. The mission of NNRI is to accelerate the translation of laboratory based research into clinical trials for treatments and cures of retinal degenerative diseases. It is a medical research institute that obtains support from government agencies, corporations and private foundations. It may also receive royalties or licensing fees from the drug discovery processes and commercialization of new therapies. Further information is available at www.nnri.info.

Sunday, May 3, 2009

Tufts Medical Center Researchers Create a New Predictive Model for Age-related Macular Degeneration (AMD)

Tufts Medical Center Researchers Create a New Predictive Model for Age-related Macular Degeneration (AMD)


BOSTON, April 23 /PRNewswire-USNewswire/ -- Researchers at Tufts Medical Center have created a formula for predicting how likely it is that individuals with certain genetic profiles and lifestyle behaviors will develop advanced Age-related Macular Degeneration (AMD), a potentially blinding condition that currently affects an estimated two million older Americans and is increasing dramatically as the population ages.

The study, led by Johanna M. Seddon, MD, ScM, Professor of Ophthalmology at Tufts University School of Medicine and Director of the Ophthalmic Epidemiology and Genetics Service at Tufts Medical Center, evaluated six genotypes that either increase or decrease risk for AMD. In addition to age, sex, and education, she also incorporated smoking status and higher body mass index (BMI) which increase risk of AMD, and supplementation with a high-dose formulation of antioxidants and zinc which delays progression of the disease. Using their new algorithm, Dr. Seddon and her colleagues determined that several genotypes plus the lifestyle factors can predict progression to the advanced forms of AMD with a certainty as high as 83%. The paper, "Prediction model for prevalence and incidence of advanced age-related macular degeneration based on genetic, demographic, and environmental variables" was published in the May issue of the journal Investigative Ophthalmology & Visual Science.

Their research also shows that although AMD has a strong genetic component, healthy behaviors can modify your genetic susceptibility. For example, among individuals with one genotype, the homozygous C3 risk genotype, the likelihood of progression to the advanced form of AMD increased from about three-fold for nonsmokers to nearly 10-fold for smokers.

"Our algorithm could help with the selection of study participants for treatment trials and could one day enable doctors to choose the most efficacious treatment for individual patients," Dr. Seddon said. "It also gives any older person concerned about AMD, or any patient with early stages or a family history of AMD, even more incentive to avoid risk factors such as smoking and excessive weight."

The study included 1,446 individuals from the Age-Related Eye Disease Study who had 6.3 years of follow-up, of which 279 progressed to the advanced stages of AMD.

About Tufts Medical Center

Tufts Medical Center is a not-for-profit, 439-bed academic medical center that is home to both a full-service hospital for adults and Floating Hospital for Children.