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Archive for the Other diseases Category

AGTC Announces Topline Interim Six-Month Data of XLRS Gene Therapy from Ongoing Phase 1/2 Clinical Trial

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Applied Genetic Technologies Corporation (AGTC), a leading developer of gene therapies, has reported the six-month follow-up data in all patients treated with its gene therapy for X-linked retinoschisis (XLRS) in its Phase 1/2 clinical trial. The company reported that no consistently significant clinical activity from the treatment was observed after six months. AGTC will continue to analyze all of the XLRS trial data and complete all visits specified in the clinical protocol. However, if the data at additional follow-up data visits are consistent with the current six-month findings, the company has stated that they will not further develop the product.
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Ophthotech to Boost Development of Gene Therapy for Best Disease

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Ophthotech Corporation, a biopharmaceutical company focused on the development of therapies for orphan retinal diseases and age-related macular degeneration, has announced an evolving, commercial partnership with the University of Pennsylvania and University of Florida to develop a gene therapy for Best disease caused by BEST1 mutations.
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Recording Available for Achromatopsia Teleconference Hosted by AGTC, Foundation, and Achroma Corp

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Achromatopsia is a challenging inherited retinal disease causing extreme light sensitivity, as well as impaired visual acuity and color perception. Approximately 75 percent of cases are caused by mutations in the genes CNGA3 or CNGB3.

Applied Genetic Technologies Corporation (AGTC), Achroma Corp, and the Foundation Fighting Blindness hosted a one-hour teleconference on the condition. The call highlighted the difficulties patients have in getting a diagnosis for achromatopsia, the importance of genetic testing, and gene therapy clinical trials underway. Presenters included Dr. Christine Kay, a clinical trial investigator with Vitreo Retinal Associates (Gainesville, Florida), and Bridget Vissari, president of Achroma Corp, which is expediting cures for achromatopsia.
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History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy

Spark LogoSpark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition.

Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. Study participants with severe vision loss reported putting away their navigational canes, seeing stars, being able to read, and recognizing faces of loved ones. Vision restoration has persisted for at least three years. The treatment is also designed to work for people with retinitis pigmentosa (RP) caused by RPE65 mutations. Continue Reading…

VISIONS 2016 – Keynote Speaker Dartanyon Crockett on Fear and Courage

Dartanyon CrockettAlmost from the start, the cards were stacked against Dartanyon Crockett. He was diagnosed, at an early age, with Leber’s disease, which caused him to be legally blind. At the age of 8, he lost his mother. And by middle school, he was relentlessly teased for being visually impaired.
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A Steady Hand in Saving Vision

Dr. Robert MacLaren performing eye surgery.A retinal researcher can develop the best vision-saving stem-cell or gene therapy ever imagined, but if the doctor or surgeon can’t deliver it to the retina safely and effectively, the treatment is worthless. It could even be harmful.
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Fighting Blindness Gets Sheepish

A sheep in tall grass.We all know from the nursery rhyme “Mary Had a Little Lamb” that “everywhere that Mary went, the lamb was sure to go.” But what if the lamb had a vision-robbing retinal disease, making it challenging to find Mary prancing around a sunlit meadow, or catching some rays on the Jersey Shore?
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ARVO 2016: High-School Sophomore Finds Gene Mutation in Family with Choroideremia

Aditya A. Guru explaining his posterWhile I was perusing posters on genetic research in the exhibit hall at the ARVO annual meeting, Radha Ayyagari, Ph.D., an FFB-funded genetic scientist from the University of California, San Diego took me by the arm and said, “Come with me. There’s something you really need to see.”
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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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Two New Videos Highlight FFB’s Vital Role in Retinal Research

Dr. Boye in her lab, conducting a tourAs everyone knows, there is never just one side to a story. That’s certainly true in the case of Dr. Shannon Boye, whose FFB-funded research is the subject of not just one but two new Foundation videos.
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