Orchard Therapeutics, Fondazione Telethon and Ospedale San Raffaele Announce Exclusive Worldwide License Agreement for the Treatment of MPS-I

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BOSTON, LONDON and MILAN, Italy, May 28, 2019 (GLOBE NEWSWIRE) -- 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 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 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 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.

The MPS-I program has shown promising clinical data in an ongoing proof-of-concept study in patients with the severe Hurler subtype, who were treated with autologous HSC gene therapy, referred to as OTL-203, using a cryopreserved formulation and a lentiviral vector.

“Developing safe and effective treatments for neurometabolic diseases has been a challenge that we believe autologous HSC gene therapy has the potential to overcome,” said Luigi Naldini, director of SR-Tiget and head of the Gene Transfer Technologies and New Gene Therapy Strategies Unit. “Preliminary data obtained to date from the ongoing clinical trial for MPS-I show signs of metabolic correction in patients with the most severe subtype of MPS-I, known as Hurler syndrome. We are pleased to continue our partnership with Orchard as we work together to develop potential HSC gene therapies to treat MPS-I and other devastating rare diseases.”

SR-Tiget was created in 1995 through a joint venture between Ospedale San Raffaele, a highly specialized internationally renowned scientific, clinical and academic facility, and Fondazione Telethon, one of the main biomedical charities in Italy focused on rare genetic diseases. SR-Tiget is now an international point of reference for gene therapy research and cell transplantation for many genetic disorders.

Orchard’s portfolio of , autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis®, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD) and transfusion-dependent beta-thalassemia (TDT), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Fondazione Telethon press office  HAVAS PR Milan Thomas Balanzoni Ph. 02 85457047 – cell. 3463204520 e-mail:   

IRCCS Ospedale San Raffaele Press officeGea Gardini – Marta AmmoniPh. 02 2643 6255/4466  - 334 6090384e-mail: web: 

More news and information about Orchard Therapeutics (Europe) Limited

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Globe Newswire: 12:00 GMT Tuesday 28th May 2019

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