Orchard Therapeutics Announces New OTL-201 Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA) Accepted for Oral Presentation at 62nd American Society of Hematology Annual Meeting

Preliminary results from first patient in ongoing proof-of-concept trial show evidence of engraftment of gene-modified cells three months following treatment

BOSTON and LONDON, Nov. 05, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today 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 three-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.

About OTL-201 and MPS-IIIA
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 buildup 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.¹ 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.