Search Results for: "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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Let’s Talk Gene Therapy Research With Dr. Mary Sunderland & Dr. Chad Andrews (VIDEO)

June 12th, 2017 by FFB Canada

On the first Tuesday of every month, the Foundation Fighting Blindness sends out our e-newsletter featuring the latest and most exciting research breakthroughs. Soon after each e-newsletter, our very own Director of Research and Education, Dr. Mary Sunderland, takes to our Facebook page to do a live broadcast discussion of the articles featured in the…

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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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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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A Sight for Sore Eyes: Using Gene Therapy and Gene Editing to Treat Blindness

April 21st, 2016 by FFB Canada

Story guest authored by Erik Fraunberger, a neuroscience graduate student at the Alberta Children’s Health Research Institute in Calgary, Alberta. We live in exciting times! With each passing day, we get one step closer to safely and effectively treating the vision diseases that affect more than a million Canadians. In recent years, scientists and clinicians have been…

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Getting XIAP Gene Therapy Ready for the Clinic

December 16th, 2015 by FFB Canada

By Dr. Mary Sunderland, FFB Director of Research & Education Dr. Cathy Tsilfidis has shown that XIAP gene therapy can protect photoreceptors. We are excited about the potential of XIAP to become a sight-saving therapy for a variety of degenerative retinal diseases. Photoreceptors are the light-sensitive cells in the eye and it is the loss…

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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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