EMA approves Groundbreaking Gene Therapy for Super Rare ‘Bubble Baby’ Disease

01/06/2016 - 3 minutes

GSK, Fondazione Telethon and Ospedale San Raffaele have announced that the EMA has approved Strimvelis for a super rare immunodeficiency disease in Children.

gsk_biotech_pharma_strimvelis_gene_therapy_ema_ada_scidFollowing 20 years of research at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Italy, this is the news patients have been waiting for.

Strimvelis is the first ex vivo stem cell gene therapy to treat patients with the very rare type of Severe Combined Immunodeficiency (SCID) due to Adenosine Deaminase deficiency (ADA-SCID).

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

The current go-to therapy for children born with ADA-SCID involves bovine enzyme replacement, such as Adagen (Pegademase Bovine) from Sigma Tau Pharmaceuticals (also in Italy).

But this requires frequent injections and is not as effective as it could be,

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