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GSK and MolMed sign immunodeficiency deal

GlaxoSmithKline and MolMed have signed a deal to develop an investigational gene therapy for ADA-SCID, the so-called ‘bubble boy disease’

GlaxoSmithKline (GSK) has signed an agreement with MolMed, under which the Italian company will develop a production process for an ex vivo gene therapy.

The gene therapy, which has been developed by the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) and licensed by GSK for development and commercialisation, is in late-stage clinical trials for the treatment of Adenosine Deaminase Deficiency - Severe Combined Immune Deficiency (ADA-SCID), an extremely rare and life-threatening condition affecting approximately 350 children globally, commonly referred to as 'bubble boy disease'. 

Patients with ADA-SCID are currently treated with bone marrow transplants or enzyme replacement therapy (ERT), but neither treatment option is ideal: transplantation requires a viable donor and runs the risk of immune rejection, and ERT requires frequent injections and treats the symptoms without curing the disease. The gene therapy therefore represents a significant advance.

Despite its low prevalence, the molecular mechanism of ADA-SCID is well understood, as it is caused by a fault in a single gene. This means that it is possible to take the patient's own stem cells from the bone marrow, treat the cells with the gene therapy and then return the treated cells to the patient.

Using a patient's own cells avoids immune compatibility issues. Additionally, phase I and II studies have demonstrated the potential of HSR-TIGET/GSK's gene therapy treatment option to restore long-term immune function and protect against severe infections in children with ADA deficiency. 

“Not only does the lead programme for ADA-SCID represent an opportunity to provide a treatment option for an under-served patient population, by combining our experience in manufacturing complex biological products with the pioneering research of world-leaders in gene and stem cell therapies, we have the chance to dramatically advance this field,” said Dr Philippe Monteyne, Head of Development and Chief Medical Officer for GSK Rare Diseases, when the therapy collaboration was first announced, in October 2009.

Under the terms of the newly-announced agreement, MolMed will receive up to €5.5m in revenues from GSK during a two-year period. 

laudio Bordignon, chairman and chief executive officer of MolMed, said: “I am very proud that today MolMed can give its industrial contribution to this innovative and advanced field of medicine.”

9th August 2011

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