Orchard Therapeutics PLC is licensing a potential gene therapy treatment for a debilitating and often deadly rare metabolic disease known as mucopolysaccharidosis type I.
Italian charity Fondazione Telethon and research institute Ospedale San Raffaele Srl have granted an exclusive worldwide license to develop, manufacture and commercialize the stem cell gene therapy OTL-203, which may treat the disease caused by the deficiency of an enzyme needed to break down polysaccharides, a type of complex carbohydrate.
The polysaccharides accumulate in tissues, resulting in symptoms that can include excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. Complications related to MPS I also include skeletal deformity, loss of vision and hearing and problems of the heart and lungs.
The San Raffaele-Telethon Institute for Gene Therapy, known as SR-Tiget, has an existing agreement with Orchard to develop treatments for metachromatic leukodystrophy, Wiskott-Aldrich syndrome, and transfusion-dependent beta-thalassemia. SR-Tiget is a joint venture between Ospedale San Raffaele and Fondazione Telethon, an Italian biomedical charity focused on rare diseases.
A proof of concept study for OTL-203 in four patients with a severe type of MPS I, known as Hurler syndrome, is ongoing. The trial expects to enroll eight patients by the first half of 2020.
Current treatment options for MPS I include bone marrow transplant and chronic enzyme replacement therapy, which has limited efficacy on the neurological symptoms. People with untreated Hurler syndrome rarely live past the age of 10.
London-based Orchard Therapeutics did not disclose the amount of the deal but said it included an up front cash payment as well as payments contingent on the treatment hitting certain milestones, plus royalties.