MolMed and Fondazione Telethon to collaborate on gene therapy for six rare diseases

Italian biotech firm MolMed SpA (Milan: MLM) and the charity Fondazione Telethon have entered into an accord to develop and manufacture novel gene therapy treatments for six rare genetic diseases that presently have no adequate form of cure.

The six diseases involved - metachromatic leukodystrophy (MLD), Wiskott-Aldrich syndrome (WAS), beta-thalassemia, mucopolysaccharidosis type I (MPS I), globoid leukodystrophy (GLD) and chronic granulomatous disorder (CGD) - are caused by a single defective gene, making it possible to develop a potential cure by inserting the correct form of the gene into the patient’s own stem cells, derived from bone marrow, through ex vivo gene transfer technology.

Under the agreement, MolMed will develop and produce clinical grade lentiviral vectors carrying the relevant therapeutic gene and manufacture patients’ cells to be investigated in clinical trials. Studies are ongoing for MLD and WAS since 2010. MolMed will be receiving up to 8.3 million euros ($11.7 million) in revenues over a four-year period.