GSK signs $300m rare disease deal with Angiochem

GlaxoSmithKline (GSK) has teamed up with Canadian biotech Angiochem to research and develop new treatments for rare diseases.

The deal, signed before tomorrow's Rare Disease Day, will cover the investigation of therapies for lysosomal storage diseases (LSDs), which include rare inherited disorders such as Fabry Disease, Gaucher's Disease and Hunter syndrome.

Angiochem will receive $31.5m in upfront cash and then further research funding and other fees could see it receive a further $300m.

The company is also eligible to receive royalties on future sales of any products commercialised as part of the collaboration.

In return, GSK will have access to Angiochem's expertise in creating compounds that can penetrate the central nervous system in the brain, restoring enzyme function.

This technique is thought to help people with LSDs, which involve gene mutations that can cause a deficiency in enzymes.

People who lack certain enzymes cannot metabolise lipids or glycoproteins within a cell, with a build up of these molecules leading to one of a variety of diseases based on the initial enzyme that is deficient.

This would improve upon current intravenous treatments for these diseases that are unable to reach the brain and restore enzyme function in the CNS, failing to improve neurological symptoms associated with LSDs such as cognitive decline and behavioural changes.

LSDs are estimate to occur in about one in every 7,000 births, with the disease mainly evident from infancy.

The deal complements GSK's own efforts into research rare disease treatments, which included setting up a specialist unit in 2010.

Other collaborative efforts from GSK into rare diseases include a potential $1.5bn deal with Isis Pharmaceuticals to develop RNA-targeted therapeutics and an agreement with Italy-based MolMed to develop a production process for an ex vivo gene therapy.

Rare Disease Day

The fifth annual Rare Disease Day, organised by the European Organisation for Rare Diseases (EURORDIS) will occur on February 29, 2012 to raise awareness for the millions of people affected by rare diseases in the world.

Events include 'Rare Diseases - a model of EU Solidarity', a European symposium to highlight the achievements of European cooperation in the field of rare diseases and to call for rare diseases to be a priority in EU public health and research policy initiatives.

EU Commissioner for Health, John Dalli, said: “In the public health agenda it is very important to ensure that enough funding is being made available for the research necessary to combat rare diseases.”