MolMed and GSK fight bubble boy
Milan/London – Italy’s MolMed SpA has signed a deal with UK-based pharmaceutical giant GlaxoSmithKline to develop a production process for a gene therapy against the rare ‘bubble boy disease’. Over the next two years, GSK will pay the Italian biotech up to EUR5.5m to work on the candidate against ADA-SCID (Adenosine Deaminase Deficiency – Severe Combined Immune Deficiency). The life-threatening disease affects approximately 350 children worldwide. ADA-SCID is caused by a mutation in a single gene that prevents the body from producing the ADA enzyme. In the treatment, which GSK acquired from San Raffaele Telethon Institute in October 2010, the faulty gene is repaired in stem cells derived from the patient’s bone marrow. Previous studies suggest the treatment is able to restore long-term immune function and protect children with ADA-SCID against severe infections. The job is very welcome at MolMed. The Milan-based biotech is still hemorrhaging money. In the first half of 2011, the firm’s EUR1.3m in revenues were dwarfed by its EUR11.8m in operating costs – and outlay won’t drop anytime soon. In late July, MolMed announced the enrolment of the first patient in a randomised Phase II trial of its investigational anticancer drug NGR-hTNF for the treatment of platinum-resistant/refractory ovarian cancer.