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24 Jun 2016 8 Comments

Bursting the Bubble: Europe’s First Gene Therapy For Kids To Be Approved By For Treating Bubble Boy Disease

GlaxoSmithKline is one step closer to receiving clearance from European Commission for its gene therapy Strimvelis to treat “Bubble Boy” disease. This treatment when approved would become Europe’s first gene therapy for children.

Bubble Boy disease occurs when a child inherits faulty genes from both parents that inhibit the proper production of white blood cells, causing severe immunodeficiency. When left untreated, children with Bubble Boy disease die within the first year.  The gene therapy Strimvelis treats patients who are unable to find a bone marrow donor.

The EU panel recommendation for clearing this therapy comes after GlaxoSmithKline’s 100% successful clinical trial with patients treated surviving the average follow-up time of seven years. The therapy works by extracting the bone marrow cells from the patients, repairing them with the necessary enzymes and reinserting them. The therapy is both process and price intensive.

GlaxoSmithKline hopes to replicate this method and also apply the gene therapy concept across other diseases.

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