Navigation Links
Penn receives prestigious national award for breakthrough in gene therapy
Date:4/19/2013

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 of Ophthalmology at the University of Pennsylvania School of Medicine, and carried out in collaboration with Penn Medicine's Albert M. Maguire, MD, and Katherine A. High, MD at The Children's Hospital of Philadelphia (CHOP), has been presented with the Distinguished Clinical Research Achievement Award, the second highest given in the CRF's Annual Top 10 Clinical Research Achievement Awards. CRF award winners are cited as the most compelling examples of scientific innovation that results from the nation's investment in clinical research that can benefit human health and welfare.

The results of the most recent phase of the study for Leber's 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 we'll move forward with this particular disease and get approval for the drug that we've 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 team's efforts to further gene therapy research," said Joan O'Brien, 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."


'/>"/>

Contact: Katie Delach
katie.delach@uphs.upenn.edu
215-349-5964
University of Pennsylvania School of Medicine
Source:Eurekalert

Related biology news :

1. Peihua Jiang receives Ajinomoto Award for Young Investigators in Gustation from AChemS
2. Forsyth Institute receives $4.1 million grant for new center
3. Einstein receives $12 million grant to develop device for preventing HIV infection
4. Wayne State vision restoration technology receives Notice of Allowance for US patent app
5. BU researcher receives highest honor from the National Birth Defects Prevention Network
6. Geneticist Svante Pääbo receives the $500,000 Gruber Genetics Prize
7. Artificial retina receives FDA approval
8. NTU Provost receives prestigious Imperial College fellowship joining the ranks of top UK scientists
9. Xenon Receives a Milestone Payment for Marketing Approval of Glybera in Europe
10. Brainwave "Balancing" Research Receives $1 Million Grant From The Susanne Marcus Collins Foundation, Inc.
11. Celladon Corporation Receives Notice of Allowance from United States Patent and Trademark Office
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/11/2017)... 2017 Research and Markets has announced the ... to their offering. ... eye tracking market to grow at a CAGR of 30.37% during ... Market 2017-2021, has been prepared based on an in-depth market analysis ... and its growth prospects over the coming years. The report also ...
(Date:4/5/2017)... , April 5, 2017  The Allen Institute ... Allen Cell Explorer: a one-of-a-kind portal and dynamic digital ... 3D imaging data, the first application of deep learning ... human stem cell lines and a growing suite of ... platform for these and future publicly available resources created ...
(Date:4/3/2017)... WASHINGTON , April 3, 2017 /PRNewswire-USNewswire/ ... single-cell precision engineering platform, detected a statistically ... cell product prior to treatment and objective ... highlight the potential to predict whether cancer ... prior to treatment, as well as to ...
Breaking Biology News(10 mins):
(Date:5/22/2017)... ... , ... Stratevi, a boutique firm that partners with healthcare companies to creatively ... office in downtown Boston at 745 Atlantic Ave. , “We are seeing that ... on the value they provide, not just to patients, but also payers. Having a ...
(Date:5/22/2017)... ... May 22, 2017 , ... Cancer diagnostics and ... booth B2 at the Association for Pathology Informatics Annual Summit at ... to demonstrating its Cancer Diagnostic Cockpit and Consultation Portal, Inspirata will present research ...
(Date:5/21/2017)... ... May 20, 2017 , ... CNSDose ... lengthy trial and error process by finding the right antidepressant faster. CNSDose ... the doctor-patient relationship through a personalized approach to treatment. , A ...
(Date:5/19/2017)... , ... May 19, 2017 , ... ... Proof-of-Concept Program. Academic researchers with technologies ripe for commercialization, and who are ... and Delaware, are encouraged to submit proposals. QED, now in its tenth round, ...
Breaking Biology Technology: