Orchard Therapeutics share their updates on their disease programs 

Orchard Therapeutics, a leading pharmaceutical company has announced progress made advancing gene therapy aimed at correcting neurometabolic diseases and has also provided reports on their first quarter 2019 financial results.

  • Earlier timeline for two disease programs; Clinical Trial in Sanfilippo Syndrome Type A (MPS-IIIA) now expected to start later this year and updates with Marketing Authorization Application (MAA) Submission for the Treatment of Metachromatic Leukodystrophy (MLD) Brought Forward to the First Half of 2020

  • Secured a worldwide license for a clinical-stage HSC gene therapy program in MPS I – New Collaboration with Fondazione Telethon and Ospedale San Raffaele for Clinical Program in Mucopolysaccharidosis Type I (MPS-I) Using Ex Vivo Autologous Hematopoietic Stem Cell Gene Therapy

  • Ended the First Quarter of 2019 with Approximately $300M in Total Cash and Investments; Newly Secured $75 Million Credit Facility Extends Runway into 2021

Summary of Neurometabolic updates

Mucopolysaccharidosis Type I (MPS-I): Orchard has been granted an exclusive worldwide license from Fondazione Telethon and Ospedale San Raffaele to research, develop, manufacture and commercialize an ex vivo autologous HSC gene therapy program for the treatment of MPS-I, which will be referred to as OTL-203. The program has currently shown encouraging preliminary data with signs of metabolic correction in patients with the most severe subtype of MPS-I, known as Hurler syndrome, in the ongoing proof-of-concept clinical trial being conducted at San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. As of the data presented 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. The trial is expected to enroll up to eight patients by the first half of 2020. The terms of the license include an upfront cash payment, success-based milestones and royalties on net sales.

Metachromatic Leukodystrophy (MLD): Orchard held a positive Marketing Authorization Application (MAA) pre-submission meeting with the European Medicines Agency (EMA) in early May. The company has brought forward the timeline for the planned submission of an MAA to the EMA for OTL-200 to the first half of 2020 and also expects to file a Biologics License Application (BLA) in the U.S. approximately one year after the MAA submission.

Sanfilippo Syndrome Type A (MPS-IIIA):  Last week, the Manchester University NHS Foundation Trust issued a statement that the Royal Manchester Children’s Hospital (RMCH) is the first in the world to treat an MPS-IIIA patient with an ex vivo HSC gene therapy. This was conducted under a “Specials” license, granted by the UK government for the use of an unlicensed pharmaceutical product in situations of high unmet need when there is no other treatment option available. Orchard holds the license to the MPS-IIIA program (OTL-201) and will support an upcoming

Read the full press release here

“With today’s announcements, including the addition of the MPS-I program, our gene therapy portfolio now includes nine programs from late pre-clinical to commercial stage, with the goal of having three more approved therapies available to transform the lives of children affected by some of these serious rare diseases in the next three years.”


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