MolMed in deal with GlaxoSmithKline to develop production process for a gene therapy

Italian biotech company MolMed (Milan: MLM) has signed an agreement with UK drugs giant GlaxoSmithKline (LSE: GSK) under which it will develop a production process for an investigational gene therapy for ADA-SCID (Adenosine Deaminase Deficiency – Severe Combined Immune Deficiency), a very rare and life-threatening disease that affects approximately 350 children worldwide. Under the terms of the deal, MolMed will receive up to 5.5 million euros ($7.8 million) in revenues over a two-year period.

ADA-SCID is a disease caused by the alteration of a single gene; it was thus possible to develop a therapy by inserting, through gene transfer technology, the correct form of the gene into the patient’s own stem cells derived from their bone marrow. This gene therapy, which is in late stage clinical trials, has been developed by Italy’s San Raffaele Telethon Institute for Gene Therapy (HSRTIGET) and was in-licensed by GSK to develop and commercialize the therapy (The Pharma Letter October 10, 2010).

Early last year, GSK set up a unit specifically dedicated to rare diseases (TPL February 5, 2010). With between 6,000 to 8,000 rare diseases, Marc Dunoyer, global head of GlaxoSmithKline Rare Diseases, said he recognizes that the unit’s efforts will need to be focused, stating: “We will target those diseases where we realistically believe we can make a significant contribution; ideally we will be creating new medicines, but importantly by adding to the weight of scientific knowledge into rare diseases to the benefit of all working in this area.”