Author Interviews, Genetic Research, Ophthalmology / 07.11.2017
FDA Advisory Committee Recommended Approval of First Gene Therapy For Inherited Eye Disease
MedicalResearch.com Interview with:
Dr. Stephen M. Rose, PhD
Chief Research Officer
Foundation Fighting Blindness
Dr. Rose discusses the FDA advisory panel unanimously recommended approval of Spark Therapeutics' Gene Therapy Luxturna for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal dystrophies, a group of rare blinding conditions caused by one of more than 220 different genes.
MedicalResearch.com: Would you tell us a little about IRD? Whom does it affect and how? How common is this disorder?
Response: The retina at the back of the eye is responsible for collecting light and turning it into signals that are transmitted to the brain and interpreted as vision. Think of the retina as the film in a camera, or more recently the sensor at the back of a digital camera. Inherited rare retinal degenerations are when the retina at the back of the eye deteriorates and loses its ability to capture light, thereby leading to blindness.
iRDs can affect anyone, no matter race or ethnicity. These are inherited conditions that are passed down from parents to children, if a parent or both parents are either affected already or are carriers for a variant in any of the over 250 genes responsible for retinal degeneration.
There are over 15 different types of iRDs, with retinitis pigmentosa being the most common with a US affected population around 100,000. The rest of the iRDs make up another approximately 100,000 affected individuals in the US, so there are about 200,000 total affected individuals in the US. Worldwide these iRDs affect somewhere around one to two million individuals.
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