GSK collaborates on rare genetic diseases
pharmafile | October 19, 2010 | News story | Research and Development | Fondazione San Raffaele, Fondazione Telethon, GlaxoSmithKline, Stem cells, gene therapy
GlaxoSmithKline has partnered with one of the biggest biomedical charities in Italy and the country’s largest private research institute to develop novel treatments for rare genetic disorders.
The strategic alliance with Fondazione Telethon and Fondazione San Raffaele will focus on gene therapy carried out on stem cells taken from the patient’s bone marrow.
Under the terms of the agreement, GSK will gain an exclusive licence to develop and commercialise an investigational gene therapy, for ADA Severe Combined Immune Deficiency (ADA-SCID) – a rare and life-threatening immune deficiency, which affects approximately 350 children worldwide.
GSK said phase I/II studies have demonstrated the potential of this treatment option to restore long-term immune function and protect against severe infections in children with adenosine deaminase (ADA) deficiency.
Dr Philippe Monteyne, head of development and chief medical officer for GSK rare diseases, said: “This alliance is an important addition to GSK’s growing portfolio in rare diseases and advances the work our stem cell researchers have been doing to deliver transformative regenerative therapies.”
Fondazione Telethon, one of the biggest biomedical charities in Italy, will receive an upfront 10 million euro payment from GSK and is eligible to receive further payments upon successful completion of a number of predetermined development milestones.
Francesca Pasinelli, general manager of Fondazione Telethon, said: “This day is one of extraordinary importance for us.
“Through the years we have been raising donations with the promise of advancing towards the cure of rare genetic diseases. With this alliance we can show that the excellent scientific research we have funded could actually lead to viable therapies available to all patients.”
Maria Grazia Roncarolo, scientific director of the San Raffaele Scientific Institute, said: “San Raffaele and Telethon have a shared ambition to translate basic discoveries in the field of molecular medicine into treatments for patients.
“We are really excited by the alliance with GSK, because we can now progress our research and know-how into potential treatments.”
The alliance capitalises on research performed at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), a joint venture between Fondazione Telethon and Fondazione San Raffaele that was established since 1995.
The deal will also see GSK co-develop with its new partners, six further applications of ex vivo stem cell therapy, using a new gene transfer technology developed by HSR-TIGET scientists, with the potential to treat a range of rare disorders.
This first of these will be metachromatic leukodystrophy, a rare and often fatal genetic neurological disorder, and Wiskott-Aldrich Syndrome, a condition with variable expression, but commonly includes immunodeficiency.
All of these disorders have a molecular mechanism that is well understood and are caused by faults in a single gene, making it possible for this ex vivo gene technology to correct the patient’s own bone marrow stem cells.
When the treated stem cells are returned to the patient they express the corrected protein, providing an opportunity to treat the disease and side step much of the risk associated with immune-incompatibility that comes from stem cells supplied by a donor.
Ben Adams
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