EU Commission approves Strimvelis to treat rare disease ADA-SCID (Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency)- GSK
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) announced that the European Commission has approved Strimvelis, the first 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). A child born 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 the first corrective gene therapy for children to be awarded regulatory approval anywhere in the world. It is indicated for the treatment of patients with ADA-SCID for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
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