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Posts tagged arvo

Forty-Four High-Impact Retinal-Research Efforts Highlighted at FFB-Casey Innovation Summit

For anyone wanting to know about the latest clinically focused research for inherited retinal diseases (IRDs), the Innovation Summit for Retinal Cell and Gene Therapy was the place to be.

In its fourth year, the meeting is becoming the world’s most comprehensive overview of the promising research underway for emerging IRD treatments. Hosted by the Foundation Fighting Blindness and Casey Eye Institute (Oregon Health & Science University), the Innovation Summit featured 44 information-packed presentations from research and industry leaders. Summit co-hosts were Casey’s Mark Pennesi, MD, PhD, and Trevor McGill, PhD, and Patricia Zilliox, PhD, chief drug development officer of FFB’s Clinical Research Institute. More than 250 researchers and industry leaders were in attendance. Spark Therapeutics was a sponsor of the event.
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Clinical Trial Authorized in the U.S. for Emerging LCA 10 Therapy

ProQR, a biotechnology company in the Netherlands, has received authorization from the U.S. Food and Drug Administration to start a Phase I/II clinical trial for its therapy known as QR-110, which is being developed for Leber congenital amaurosis type 10 (LCA 10). The genetic retinal condition causes severe vision loss in children. QR-110 targets the specific mutation p.Cys998X in the CEP290 gene, also known as c.2991+1655A>G mutation. It is estimated that it affects about 2,000 people in the Western world.

QR-110 was a featured topic at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, May 7-11. About 12,000 eye researchers and industry professionals attended the event.
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ARVO 2016: ProgSTAR, FFB-CRI’s Stargardt Disease Patient Study, Highlighted

Janet Cheetham, ProgSTAR's liaison to FFBOne of the hot topics at ARVO 2016 is ProgSTAR, the natural history study for people with Stargardt disease funded by the Foundation Fighting Blindness Clinical Research Institute (FFB-CRI). I caught up with Janet Cheetham, Pharm.D., the project’s liaison to FFB, to explain why the effort is important to therapy development. Having spent more than three decades in the development of retinal and ophthalmological treatments at Allergan, she brings a wealth of insight and knowledge to her role.
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ARVO 2016: Choroideremia Gene Therapy in Clinical Trial Continues to Perform Well

Dr. Robert MacLaren during surgery. The annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Seattle won’t start for another three days, but already there’s exciting research news to report. Five of six patients in NightStaRx’s choroideremia gene-therapy trial at the University of Oxford in the United Kingdom, which began in 2013, continue to benefit from the treatment.
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ARVO 2015 Highlight: The National Eye Institute Invests $4 Million in Audacious-Goals Research

future of retinal researchThe National Eye Institute’s (NEI) establishment of its Audacious Goals in 2013 was a watershed moment in the drive to cure blinding retinal diseases. The mission of the program—to regenerate the neurons and neural connections in the eye and visual system—is synonymous with the Foundation’s mission to eradicate retinal diseases. Most important, it means that the NEI is making significant investments in research that will benefit people with retinal conditions, such as retinitis pigmentosa, Usher syndrome, Stargardt disease, and age-related macular degeneration.
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ARVO 2015 Highlight: AMD Gene Therapy Performs Encouragingly in Human Study

an AMD eyeWhile treatments such as Lucentis®, Avastin®, and Eylea® have been saving and restoring vision for people with wet age-related macular degeneration (AMD) over the last several years, they have a significant drawback: The therapies require regular injections into the eye—in some cases, monthly—for the life of the patient.
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ARVO 2015 Highlight: A Cut-and-Paste Approach to Fixing Retinal-Disease Genes

gene editingI just returned from the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), the world’s largest eye-research conference, held this year in Denver. It attracted more than 11,000 scientists and physicians, including many of the 187 retinal researchers funded by the Foundation. The FFB science team and I worked feverishly to learn as much as possible about the latest news from the retinal-research front. It was truly exhilarating—albeit, at times, overwhelming.
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ARVO 2014: LCA Gene Therapy Recipient Featured During Keynote

Yannick Duwe and his father, Tony, at ARVOGene-therapy pioneer Jean Bennett, M.D., Ph.D., may have been the keynote speaker for the closing session at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), but Yannick Duwe, a 15-year-old patient with Leber congenital amaurosis (LCA) who was treated in her clinical trial six years ago, stole the show. He and his father, Tony, were part of a panel which also included members of Dr. Bennett’s research team.
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ARVO 2014: European Collaboration Developing Cross-Cutting, Vision-Saving Therapies

The DRUGSFORD collaborativeOne of the most intriguing reports on therapy development at the annual meeting of the Association for Research in Vision and Ophthalmology came from a European collaborative project called DRUGSFORD, which is derived from the phrase “drugs for retinal degeneration.” With funding from the European Union, the project brings together three research groups and two companies to develop a therapy that can treat a disease pathway common to many retinal degenerations. Simply put, they’re creating therapies that can save vision in as many people as possible, independent of the genetic cause of disease.
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ARVO 2014: Three Promising CEP290 Gene Therapy Alternatives

Renee Ryalls explains the dual-AAV gene therapy she's developing.While gene therapies for retinal degenerative diseases are making groundbreaking strides in both human and laboratory studies, the most widely and successfully used human-engineered virus for delivering replacement genes to retinal cells — the adeno-associated virus, or AAV — has one significant limitation. It can’t deliver relatively large genes, namely those larger than about 4.5 or 5 kilobases (kb). (Bases are the building blocks of a gene, and its size is expressed in kilobases.)
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