IRDs -- or inherited retinal dystrophies -- put thousands of people at risk for blindness. IRD is a catchall term for a host of problems affecting the iris. Spark Therapeutics has been pursuing a solution to problems triggered by the gene RPE65 with its drug SPK-RPE65, a gene therapy. The company just announced that positive Phase 3 data demonstrates SPK-RPE65's effectiveness in improving vision among test subjects with IRDs, with no adverse effects reported.
The treatment inserts corrective genes into cells that aren't operating properly due to mutations. Study results showed "the intervention group achieved statistically significant improvement over control subjects in both functional vision and light sensitivity." Spark delivers results that are especially heart-warming when it comes to children: "following a single injection of SPK-RPE65 in one eye, the children from our Phase 1 trial no longer depended on visual aids to carry out classroom activities and were able to navigate and play more like normally sighted children." People with eyes on the stock market are also interested. Spark Therapeutics has jumped significantly with the news.
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