Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation

Orchard Therapeutics recently announced the presentation of new clinical data at the upcoming 62^nd American Society of Hematology (ASH) Annual Meeting to be held virtually December 5-8, 2020. The oral presentation will highlight data from the first patient treated in the ongoing proof-of-concept study of OTL-201, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy being studied for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA).

“MPS-IIIA is a progressive, life-threatening metabolic disease with no approved treatment options,” said Professor Robert Wynn, Chief Investigator at The Royal Manchester Children’s Hospital, part of Manchester University NHS Foundation Trust. “We are pleased to see encouraging initial results, including evidence of engraftment of gene-modified cells, an important first step in the investigation of whether OTL-201 could address critical unmet needs for patients with MPS-IIIA. We look forward to continuing to advance this program to add to the growing body of evidence supporting the use of HSC gene therapy to treat severe neurometabolic conditions.”

Preliminary results from the first patient treated with OTL-201 show evidence of engraftment of gene-modified cells, supra-physiological N-sulphoglucosamine sulphohydrolase (SGSH) enzyme expression in multiple lineages, and reduction of heparan sulfate in plasma, cerebrospinal fluid, and urine over an initial 3-month follow-up period. Additional follow-up data and an update on the trial status will be shared at the time of the oral presentation.

Oral Presentation Details

Ex-Vivo Autologous Stem Cell Gene Therapy Clinical Trial for Mucopolysaccharidosis Type IIIA: Trial in Progress – NCT04201405
Publication number: 676
Session: 801. Gene Editing, Therapy and Transfer I
Date and time: Monday, December 7, 2020; 12:45 p.m. PT
Abstracts are available online at the ASH Annual Meeting website.

Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in the N-sulphoglucosamine sulphohydrolase (SGSH) gene, which, when healthy, helps the body break down sugar molecules called mucopolysaccharides, including heparan sulfate. The build-up of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years. There are currently no approved treatment options for MPS-IIIA. OTL-201 is an investigational ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-IIIA. It uses a modified virus to insert a functional copy of the SGSH gene into a patient’s cells.

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist. Orchard has its global headquarters in London and US headquarters in Boston. For more information, visit www.orchard-tx.com.

Manchester University NHS Foundation Trust is one of the largest NHS trusts in England and a leading provider of specialist healthcare services. Its nine hospitals are home to hundreds of world class clinicians and academic staff committed to finding patients the best care and treatments. For more information, visit www.mft.nhs.uk.