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Gene therapy has come of age over the past few years. And Johnson & Johnson this morning gave its biggest signal yet that it wants in on the emerging field.
— xconomy new york. Ben Fidler. January 31, 2019
The deal comes at a time in which gene therapies have, at long last, made their way to the U.S. markets. In 2017, the FDA approved the first treatment to fix a faulty gene with a patient’s body— voretigene neparvovec (Luxturna), from Spark Therapeutics (NASDAQ: ONCE), for a rare form of inherited blindness. Another, known as Zolgensma, from Novartis (NYSE: NVS), could be approved in the U.S. this year, for the rare disease spinal muscular atrophy. And others are making clinical progress in blood diseases like hemophilia and beta-thalassemia, rare disorders such as Duchenne muscular dystrophy, and more. Two gene therapies were approved earlier in Europe.

These developments are the culmination of decades of scientific research on how to effectively and safely deliver genes to cells. Gene therapies shuttle DNA material into the body, typically with the help of an engineered virus, to help the body produce a critical protein—in perpetuity, theoretically. In hemophilia patients, for instance, experimental gene therapies help people make proteins that clot blood. Luxturna helps patients produce a protein that makes light receptors work in the eye.

With $100M MeiraGTx Deal, J&J Signals Bigger Move Into Gene Therapy

Good article from Xconomy site (Author: Ben Fidler) about J&J and field of gene therapy. You can read it HERE.