EC OKs GSK’s Rare Disease Gene Therapy

GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) have received marketing approval from the European Commission (EC) for Strimvelis, an ex-vivo stem-cell gene therapy to treat patients with a very rare disease called ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency).

ADA-SCID affects an estimated 15 children per year in Europe and following the EC’s pproval, patients with the condition who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele in Milan.

ADA-SCID is a very rare disorder caused by a faulty gene inherited from both parents. This faulty gene stops the production of an essential protein called adenosine deaminase (ADA), which is required for the production of lymphocytes (a type of white blood cell). A child with ADA-SCID does not have a healthy, fully-functioning immune system and as a consequence, is unable to fight off everyday infections. Strimvelis (autologous CD34+ cells transduced to express ADA) is a corrective gene therapy indicated for the treatment of patients with ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.  

Source: GlaxoSmithKline 

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