Gene Therapy Approvals for a Growing List of Diseases

Advancement of gene therapy for rare and life-threatening diseases is likely to prove to be one of the greatest medical advances in the coming years, says RNCOS.

According to a new research report by RNCOS entitled, ?Global Global & US Gene Therapy Market Forecast to 2020?, the advancement of gene therapy for rare and life-threatening diseases is likely to be one of the greatest medical advances in the coming years, as industry participants are increasingly strengthening the research-based approach to develop newer products.

For instance, in 2017, researchers announced that a teenage boy in France had been cured of sickle-cell disease after receiving an experimental gene therapy developed by Bluebird Bio. Sickle-cell disease is an inherited blood disorder that affects 100,000 people in the US and millions around the world. In the same year, the USFDA approved two pioneering treatments, Called CAR-T therapies, Kymriah and Yescarta that use a patient?s own immune cells to fight rare types of cancer. Kymriah treats a bone marrow cancer that affects children and young adults, and Yescarta treats a type of lymphoma. Furthermore, a boy suffering from epidermolysis bullosa got new skin created with gene therapy at a hospital in Germany. In December 2017, the FDA approved the first gene therapy for an inherited disease. The treatment, called Luxturna, manufactured by Spark Therapeutics, aims to correct a mutation responsible for a range of retinal diseases that make people gradually go blind. In human tests, the treatment has restored vision for more than two dozen patients who were losing their sight.

A gene therapy for Severe combined immunodeficiency, also known as SCID, or ?bubble boy disease,? is already available in Europe, marketed as Strimvelis by GlaxoSmithKline. SCID is a rare, inherited disorder that renders the immune system virtually useless against pathogens. BioMarin is another company working on a gene therapy that replaces the faulty gene involved in the most common type of hemophilia, effectively curing the disorder. In December, the company published early clinical trial results showing that nine patients who received its therapy saw substantial increases in the blood-clotting proteins absent in hemophilia. Meanwhile, a handful of patients with hemophilia B, a rare form of the disease, are already experiencing amazing cures after one-time treatments.

Researchers have announced some miraculous cures of patients with such rare and life-threatening diseases who were treated with gene therapies.

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