ADA-SCID is a disease caused by the alteration of a single gene. A therapy has been developed by inserting, through gene transfer technology, the correct form of the gene into patients' own stem cells derived from their bone marrow. This gene therapy, which is in late stage clinical trials, has been developed by the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) and was in-licensed by GSK.
Claudio Bordignon, chairman and chief executive officer of MolMed, commented, "We are extremely pleased to have entered into this agreement with GSK. MolMed has expertise and know-how in the field of gene and cell therapy and this agreement with GSK is an important step on the way to provide gene therapy for patients with rare diseases. I am very proud that today MolMed can give its industrial contribution to this innovative and advanced field of medicine."