Correction of ADA-SCID
by stem cell gene therapy combined with nonmyeloablative conditioning.
affects an estimated 15 children per year in Europe and following todays approval, patients with the condition who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele in Milan.
To date, over 40 ADA-SCID
patients have been treated in clinical trials with autologous ex-vivo lentiviral gene therapy at UCLA, Los Angeles and at the Great Ormond Street Hospital (GOSH) in London, UK.
The gene therapy for the treatment of ADA-SCID
was originally developed by Ospedale San Raffaele (OSR) and Fondazione Telethon (Telethon), through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and was taken forward by GSK through a strategic collaboration formed in 2010 between GSK, OSR and Telethon.
is a very rare and life-threatening disease caused by the alteration of a single gene and affects approximately 350 children worldwide.
MI) announced today the signing of an agreement with GlaxoSmithKline (GSK)(LSE:GSK) under which MolMed will develop a production process for an investigational gene therapy for ADA-SCID
(Adenosine Deaminase Deficiency - Severe Combined Immune Deficiency).