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GSK collaborates with Isis on rare diseases

GlaxoSmithKline has entered into a potential $1.5bn deal with Isis Pharmaceuticals to develop new drugs for rare and serious diseases

GlaxoSmithKline (GSK) has entered into a potential $1.5bn deal with Isis Pharmaceuticals to develop new drugs for rare and serious diseases. The alliance will make use of Isis' antisense drug discovery platform.

The deal will give GSK access to Isis' expertise in drug discovery and development of RNA-targeted therapeutics.

The agreement covers six programmes and will see Isis receive an upfront payment of $35m from GSK followed by a potential further $20m per programme (up to the phase II proof of concept stage) in milestone payments.

At the proof of concept, GSK will be given the option to license compounds thereby being responsible for all further development and commercialisation.

If all six programmes are successfully developed and commericalised for one or more indication, Isis is eligible to receive combined license fees and milestone payments worth almost $1.5bn. Isis will also receive royalties on sales from any successfully commercialised products.

"As a platform, the Isis antisense approach offers us an exciting opportunity to target certain severe diseases in a way that has not previously been possible," said Dr Patrick Vallance, senior vice-president and head of drug discovery at GSK. "Isis Pharmaceuticals is a leader in antisense technology, and this new alliance will enhance our discovery platform in this promising research area," he continued.

Antisense therapies target the proteins involved in disease processes through the RNA that is involved in building these proteins. The Isis discovery platform develops specific therapies that bind to messenger RNA (mRNA) and inhibit the production of disease-causing proteins.

Most medicines are small molecules or biologics that target a specific protein in a disease process, wheras antisense therapies prevent protein synthesis by eliminating the mRNA. This means that RNA-targeted therapeutics, or antisense therapies, represent an opportunity for a new drug class.

1st April 2010

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