In October last year, GSK linked up with San Raffaele Telethon Institute, gaining an exclusive license to an investigational gene therapy for ADA-SCID (adenosine deaminase deficiency – severe combined immune deficiency), commonly referred to as ‘bubble boy disease’. Now the drugs giant has signed a two-year, 5.5 million euro deal with MolMed which will develop a production process for the gene therapy which is in late-stage trials.
ADA-SCID is caused by a mutation in a single gene which prevents the body from producing the ADA enzyme. However, it has been 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.
The disease affects around 350 children worldwide. Patients are unable to mount their own defense against foreign organisms so without specialist intervention are at risk from life threatening infections. A bone marrow transplant (if a suitable donor can be found) is currently the best treatment option available but because gene therapy uses the patient’s own cells there is much less risk of immune rejection.