GSK, Fondazione, Ospedale Make EU Submission for Gene Therapy to Treat ADA-SCID
May 05 2015 - 6:01AM
Dow Jones News
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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