Orchard Therapeutics announces presentation of clinical data for OTL-200
Orchard Therapeutics announced the presentation of new clinical data at the upcoming European Society of Gene & Cell Therapy Annual Congress in Barcelona, Spain, October 22-25, 2019. The poster presentation will highlight initial results from the ongoing clinical trial with a cryopreserved formulation of OTL-200 for metachromatic leukodystrophy that is being conducted at the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy. Poster presentation details for OTL-200: Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Preliminary results from a clinical trial with a cryopreserved formulation of OTL-200. Metachromatic leukodystrophy is a rare and life-threatening inherited disease of the body's metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD.