Rare Daily Staff

Orchard Therapeutics said it has obtained an exclusive worldwide licensed for rights from Fondazione Telethon and Ospedale San Raffaele for an ex vivo autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of mucopolysaccharidosis type I (MPS-I), a rare and deadly lysosomal storage disorder.

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. Treatment options today include bone marrow transplant and chronic enzyme replacement therapy, both of which have limitations.

The MPS-I program, referred to as OTL-203, uses a cryopreserved formulation and a lentiviral vector. Developed by the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Italy, it is currently enrolling patients in an ongoing proof-of-concept study.

Data presented by SR-Tiget at the American Society of Gene & Cell Therapy (ASGCT) annual meeting in April 2019, reported four patients have been enrolled in the trial with follow-up of up to nine months. The therapy has shown promising clinical data in an ongoing proof-of-concept study in patients with the severe Hurler subtype, who were treated with ex vivo autologous HSC gene therapy, referred to as OTL-203, using a cryopreserved formulation and a lentiviral vector.

“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 CEO of Orchard. “This program leverages our expertise in developing ex vivo autologous HSC gene therapy candidates for neurometabolic disorders.”

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.

Photo: Mark Rothera, president and chief executive officer of Orchard

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