GSK GSK plc

By Razak Musah Baba

LONDON--GlaxoSmithKline PLC (GSK.LN), Fondazione Telethon and Ospedale San Raffaele have submitted E.U. regulatory submission for gene therapy to treat rare disease ADA-SCID.

The submission is for a marketing application to the European Medicines Agency for a gene therapy (GSK2696273) to treat patients with a rare disease. Adenosine deaminase (ADA) deficiency is an inherited disorder that causes severe combined immunodeficiency syndrome (SCID). No suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.

ADA-SCID is an incredibly rare disease in which patients cannot make lymphocytes (a type of white blood cell) and as a result have a severely deficient immune system.

GSK2696273 is an investigational gene therapy which is not approved for use anywhere in the world.

The marketing application follows on from a strategic alliance formed in 2010 between GSK, Ospedale and Telethon.

-Write to Razak Musah Baba at razak.baba@wsj.com; Twitter: @Raztweet

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