Glaxo Smith Kline is
close to seeking European approval for a gene therapy drug to
fight the immune deficiency disorder known as "bubble boy"
disease, in the latest sign of a renaissance in the technology
to fix faulty genes.
GSK will be the first big pharmaceutical company to file for
marketing approval for a gene therapy when it submits its drug
for ADA Severe Combined Immune Deficiency (ADA-SCID) in Europe
imminently, according to people familiar with the situation.
The British company has so far said little about its
program, beyond the fact that a filing is possible in 2015.
ADA-SCID affects around 350 children worldwide, leaving
sufferers extremely vulnerable to infection. Some live in a
plastic, germ-free chamber. The disorder become widely known in
the 1970s and 1980s when the media nicknamed a prominent
sufferer, David Vetter, as the "bubble boy".
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