Search Results for: spark gene therapy

First gene therapy for a blinding eye disease receives FDA approval

December 19th, 2017 by Dr. Mary Sunderland

Today we are celebrating the FDA’s pathbreaking approval of Luxturna. We have been following the development of Luxturna closely. Today’s landmark FDA approval makes Luxturna the first gene therapy approved in the US that targets a disease caused by mutations in a specific gene. The next steps involve determining the price for Luxturna, which poses…

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FDA Panel Votes Unanimously to Approve New Gene Therapy

October 13th, 2017 by Dr. Mary Sunderland

History was made on World Sight Day, October 12, 2017, when an FDA advisory panel voted unanimously to approve a new gene therapy, Luxturna, designed to treat inherited blinding eye diseases caused by mutations in the RPE65 gene. We have been following this story closely over the past few years and celebrating every milestone along…

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Gene Therapy Improves Vision in Landmark Clinical Trial

July 14th, 2017 by Dr. Mary Sunderland

Yesterday, a landmark publication in The Lancet, a prestigious journal, showed that gene therapy can improve vision in people living with a rare blinding eye disease. The study examined the effects of an experimental gene therapy – voretigene neparvovec – to treat an inherited blinding eye disease caused by mutations in the RPE65 gene. RPE65…

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Clinical Trial Updates: Gene Therapy

June 6th, 2017 by FFB Canada

Many experts agree that gene replacement therapy is a novel approach to treating inherited degenerative retinal diseases, especially when the disease is caused by a single gene defect (or mutation). Gene replacement therapies are sometimes described as “gene addition” or “gene augmentation” therapies because the treatment strategy involves replacing a damaged gene with a new,…

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Will Gene Therapies Win the Race to Restore Sight?

June 1st, 2017 by FFB Canada

There is a lot of optimism for the potential to treat inherited eye diseases with gene therapy. Around the world, scientists are exploring ways to fix the genetic errors that lead to gene-based diseases such as retinitis pigmentosa (RP) and Leber’s congenital amaurosis (LCA), editing or creating from scratch the genetic instructions that make sight…

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Experimental Gene Therapy Completes Critical Regulatory Step

May 26th, 2017 by FFB Canada

For a long time, gene therapy—the idea that genes could be used as a medical treatment—was just that: an idea. Last week, that idea took a leap forward towards becoming a reality when the company, Spark Therapeutics, completed a key application to the Food and Drug Administration (FDA). If successful, this application would make Spark’s…

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Gene Therapy for Blinding Eye Disease Poised for Government Approval

October 5th, 2015 by FFB Canada

Today is an exciting day for gene therapy! Indeed, it’s a groundbreaking day because we are celebrating the successful results of the first phase 3 gene therapy clinical trial to treat a blinding eye disease. These results are making headlines because it means that this treatment is now poised to become the first-ever gene therapy…

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RPE65 Gene Therapy Trials for Leber Congenital Amaurosis Update

May 27th, 2014 by FFB Canada

At the Association of Vision Research and Ophthalmology meeting earlier this month, Dr. Jean Bennett delivered a keynote address focusing on gene therapies. Dr. Bennett has been the principal investigator for some of the trials of gene therapy for Leber congenital amaurosis (LCA) type 2 (caused by mutations in RPE65). It is a series of…

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Four Vision Research and Treatment Trends to Watch in 2018   

January 22nd, 2018 by FFB Canada

With the December 2017 announcement that the world’s first ocular gene therapy had received approval by the U. S. Food and Drug Administration (FDA), there’s no doubt that 2017 was a landmark year for gene therapy. Looking back, 2017 was a year of firsts, including the first stem cell therapy clinical trial for macular degeneration…

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Top 10 Things We Learned at Vision Quest 2017

November 7th, 2017 by Dr. Mary Sunderland

Vision Quest is the FFB’s educational program series that brings trusted information about emerging sight-saving treatments to people living with vision loss. This year, Vision Quest brought together more than 860 people at 9 different events across Canada. The entire FFB staff participates in Vision Quest because it gives us the opportunity to learn directly…

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