Orchard Therapeutics gets license to develop gene therapy treatment

Orchard Therapeutics (Nasdaq: ORTX), Fondazione Telethon and Ospedale San Raffaele today announced that Orchard has been granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the ex vivo autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of Mucopolysaccharidosis Type I (MPS-I) developed by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The clinical-stage program is currently enrolling patients in an ongoing proof-of-concept study. As of data presented by SR-Tiget at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in April 2019, four patients have been enrolled in the trial with follow-up of up to nine months.
MPS-I is a progressive, debilitating and often life-threatening inherited lysosomal storage disorder. Patients often experience neurocognitive impairment, skeletal deformity, loss of vision and hearing, and cardiovascular and pulmonary complications, with the most severe form known as Hurler syndrome.
“We believe the use of ex vivo autologous hematopoietic stem cell gene therapy in MPS-I has the potential to fundamentally change the lives of patients born with this devastating and rapidly progressive condition,” said Mark Rothera, president and chief executive officer of Orchard. “This program leverages our expertise in developing ex vivo autologous HSC gene therapy candidates for neurometabolic disorders. Given the transformative potential seen in our MLD program, we hope we will be able to correct multiple aspects of the condition, including central nervous system dysfunction.”
The terms of the deal include an upfront payment in cash as well as contingent payments on the achievement of future development, regulatory and sales milestones, as well as royalty payments on net sales.
https://www.globenewswire.com/news-release/2019/05/28/1851980/0/en/Orchard-Therapeutics-Fondazione-Telethon-and-Ospedale-San-Raffaele-Announce-Exclusive-Worldwide-License-Agreement-for-the-Treatment-of-MPS-I.html