Gene Therapy for Rare Blindness gets FDA Approval

Gene Therapy for Rare Blindness gets FDA Approval

US Food and Drug Administration (FDA) on December 21, 2017, approved a novel gene therapy for treatment of children and adult patients with an inherited vision loss.

The new gene therapy named as Luxturna (voretigene neparvovec-rzyl) is a treatment for retinal dystrophy caused due to specific genetic mutation. “The approval marks another first in the field of gene therapy both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases,” said Scott Gottlieb, Commissioner at FDA.

‘Biallelic RPE65’ mutation-associated retinal dystrophy leads to vision loss and may cause blindness in patients. The RPE65 gene regulates function of an enzyme essential for normal vision. Mutations in the RPE65 gene results into low levels of RPE65 activity, which blocks the visual cycle leading to impaired vision. Luxturna delivers a normal copy of the RPE65 gene directly to retinal cells, which can produce the normal protein required to converts the light in to an electrical signal in the retina for restoring vision loss.

According to Advanced Therapy Products Market report published by Coherent Market Insights, gene therapy can aid in treatment of genetically inherited disorders by replacing defective or impaired gene with functional one to restore the normal functions of body. Gene therapy is advised for only those patients who have viable retinal cells as determined by the physicians. The approval of Luxturna to was granted to Spark Therapeutics Inc. “Patients with ‘biallelic RPE65’ mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed,” added Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER).

Request Sample copy of this Research Report @ https://www.coherentmarketinsights.com/insight/request-sample/1101

You may be interested

Machine Learning-Based Smartphone App to Evaluate Parkinson’s Symptoms
Information Technology and Gadgets
Information Technology and Gadgets

Machine Learning-Based Smartphone App to Evaluate Parkinson’s Symptoms

Mohit Joshi - September 21, 2018

New smartphone app HopkinsPD, utilizes the smartphone's microphone, touchscreen, and accelerometer to examine patients for symptoms while they perform daily tasks Parkinson’s affects central nervous system of…

Depressive Thoughts Affect Working Memory
Healthcare
Healthcare

Depressive Thoughts Affect Working Memory

Mohit Joshi - September 20, 2018

Researchers establish a link between prolonged depression and reduced working memory, according to a new study published in March 2018. Researchers reveal that dysphoria, which is a…

Myeloproliferative Neoplasms Treatment Market – Current Trends and Future Opportunities by 2026
Healthcare
Healthcare

Myeloproliferative Neoplasms Treatment Market – Current Trends and Future Opportunities by 2026

Albert R - September 20, 2018

Coherent Market Insights added Latest Research Report titled “Myeloproliferative Neoplasms Treatment Market Insights by Manufacturers, Growth, Trends, Regions, Product Type and Application, Geography, Forecast to 2026” to…

Most from this category