PHILADELPHIA A gene therapy study focused on finding a cure for a rare congenital blinding disease has been recognized as one of the ten most outstanding clinical research projects of the year by the Clinical Research Forum (CRF). The study, led by Jean Bennett, MD, Phd, F.M. Kirby professor ofOphthalmologyat the University of Pennsylvania School of Medicine, and carried out in collaboration with Penn Medicines Albert M. Maguire, MD, and Katherine A. High, MD at The Childrens Hospital of Philadelphia (CHOP), has been presented with the Distinguished Clinical Research Achievement Award, the second highest given in the CRFs Annual Top 10 Clinical Research Achievement Awards. CRF award winners are cited as the most compelling examples of scientific innovation that results from the nations investment in clinical research that can benefit human health and welfare.

The results of the most recent phase of the study for Lebers Congenital Amaurosis (LCA) at CHOP have led to the first Phase 3 gene therapy study in the United States and the first Phase 3 gene therapy study in the world for a non-lethal disorder. The team of researchers hopes that the studies could lead to the first approved gene therapy product in the United States.

The data from our study has already been used to develop additional clinical trials for other blinding diseases, said Bennett. There are two things that I think are really going to be important from this work: one, that well move forward with this particular disease and get approval for the drug that weve been developing, and two, that this could ultimately lead to approved treatments for other currently untreatable conditions.

Published in 2012, the winning studies are the latest in a long tradition of notable health advances that have occurred through clinical research such as eliminating polio, reducing the mortality of AIDS, and improving cancer survival rates that were propelled by combined investment in basic science and clinical research.

These patients, once rendered blind by LCA, have had their lives transformed and their vision restored by this teams efforts to further gene therapy research, said Joan OBrien, MD, chair of the department of Ophthalmology at the Scheie Eye Institute at Penn Medicine. The groundbreaking work sets the stage for the treatment of numerous other blinding conditions, but is also a shining example of what scientists with NIH resources can accomplish for the betterment of humanity.

The Clinical Research Forum is an organization comprised of the nations most prestigious and acclaimed academic medical centers and healthcare systems whose goal is to sustain and expand a cadre of talented, well-trained clinical investigators at all stage of career development, and support nurturing environments and comprehensive research capabilities within academic institutions. Its mission is to provide leadership to the national clinical and translational research enterprise and promote understanding and support for clinical research and its impact on health.

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Penn Medicine is one of the world's leading academic medical centers, dedicated to the related missions of medical education, biomedical research, and excellence in patient care. Penn Medicine consists of the Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania (founded in 1765 as the nation's first medical school) and the University of Pennsylvania Health System, which together form a $4.3 billion enterprise.

The Perelman School of Medicine has been ranked among the top five medical schools in the United States for the past 16 years, according to U.S. News & World Report's survey of research-oriented medical schools. The School is consistently among the nation's top recipients of funding from the National Institutes of Health, with $398 million awarded in the 2012 fiscal year.

The University of Pennsylvania Health System's patient care facilities include: The Hospital of the University of Pennsylvania -- recognized as one of the nation's top "Honor Roll" hospitals by U.S. News & World Report; Penn Presbyterian Medical Center; and Pennsylvania Hospital -- the nation's first hospital, founded in 1751. Penn Medicine also includes additional patient care facilities and services throughout the Philadelphia region.

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Penn Researcher Receives Prestigious National Clinical Research Award for Breakthrough in Gene Therapy

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