2017 Could Be The Year We Will Reverse Blindness
There is medicine or forms of operation today that tackle the problem of various cases blindness, but hereditary blindness had no known cure. That was until now when a new form of gene therapy can male hereditary retinal dystrophies a thing of the past.
Spark Therapeutics, a Philadelphia-based drug company, announced the launch of SPK-RPE65 in 2017. This is a gene therapy that specifically targets the inherent mutation directly responsible for blindness. The way it works is that new cells will be injected in order to correct the genetic mutation, and will be able to address and resolve the many strains of retinal dystrophies. SPK-RPE65 will be administered during a 45-minute surgical procedure, with the results presenting themselves within 30 days.
Katherine High, the company;s co-founder, said that they will complete their application with the FDA by early in 2017. Similar gene therapies like Strimvelis and Glybera are already in use in Europe, but in the US, the FDA required further clinical trials to prove its effectiveness.
The biggest problem is that gene therapy is still surrounded by a cloud of controversy after a man died because of it back in 1999. The patient went through a severe immune reaction after one such gene therapy. However, High insists that the company has since resolved those problems. She stated that the problem originated as a result of insufficient modelling through animal testing.
Spark presented their success with their SPK-RPE65 therapy at the American Academy of Ophthalmology meeting. From the 29 participants, 27 of them experienced an increase in visual functions. Furthermore, no adverse effects were observed in their patients. Gene therapy as a whole is a field that is under continuous development and future breakthroughs are sure to come. They are a key in the betterment of many lives all around the world.