Seven abstracts accepted show the potential of HSC gene therapy to transform the treatment of multiple severe rare disorders
First peer-reviewed presentation from company’s work in transduction enhancers showcases promise to improve manufacturing efficiency of lentiviral-based gene therapy
BOSTON and LONDON, April 28, 2021 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of seven abstracts at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) taking place virtually from May 11-14. Accepted abstracts include clinical data from several of its hematopoietic stem cell (HSC) gene therapy programs, including: OTL-203, being investigated for the treatment of mucopolysaccharidosis type I (MPS-I), and OTL-101, being investigated for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), as well as the company’s efforts to improve efficiency of HSC gene therapy manufacturing though the development of proprietary transduction enhancers.
“Our significant presence at ASGCT this year showcases not only the depth and breadth of our HSC gene therapy pipeline and the strength of our clinical data but also how we are using innovation to improve the way our HSC gene therapies will be manufactured in the future,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “These data demonstrate the transformative potential of HSC gene therapy and, along with our partners, we look forward to sharing results with the broader scientific and medical communities.”
The presentations are listed below, and the full preliminary program is available online on the ASGCT website.
Oral Presentation Details:
Development of an optimized lentiviral transduction process for ex vivo CD34+ hematopoietic stem cell gene therapy drug product manufacture
Abstract Number: 1
Presenting Author: Pervinder Sagoo, Ph.D., Orchard Therapeutics
Date/Time: Tuesday, May 11 at 5:30 p.m. EDT
Autologous ex vivo lentiviral gene therapy for the treatment of ADA-SCID
Abstract Number: 40
Presenting Author: Claire Booth, Ph.D., UCL GOSH Institute of Child Health
Date/Time: Tuesday, May 11 at 6:30 p.m. EDT
Immune reconstitution in transfusion dependent beta-thalassemia patients treated with hematopoietic stem cell gene therapy
Abstract Number: 79
Presenting Author: Samantha Scaramuzza, Ph.D., San Raffaele Telethon Institute for Gene Therapy
Date/Time: Wednesday, May 12 at 5:45 p.m. EDT
Hematopoietic reconstitution and lineage commitment in HSC gene therapy patients are influenced by the disease background
Abstract Number: 82
Presenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene Therapy
Date/Time: Wednesday, May 12 at 6:30 p.m. EDT
Liquid-biopsy-integration-site-sequencing allows safety studies and longitudinal monitoring of vector integration sites in LV and AAV-based in-vivo GT applications
Abstract Number: 250
Presenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene Therapy
Date/Time: Friday, May 14 at 12:15 p.m. EDT
Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome): An interim analysis with a median follow up of 19 months
Abstract Number: 219
Presenting Author: Bernhard Gentner, M.D., San Raffaele Telethon Institute for Gene Therapy
Date/Time: Friday, May 14 at 1:15 p.m. EDT
Poster Presentation Details:
Dissecting bone remodeling mechanisms and hematopoietic stem cell gene therapy impact in mucopolysaccharidosis type I Hurler bone defects
Abstract Number: 609
Presenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene Therapy
Date/Time: Tuesday, May 11 at 8:00 a.m. EDT
About Orchard Therapeutics
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.
Availability of Other Information About Orchard
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Other risks and uncertainties faced by Orchard include those identified under the heading “Risk Factors” in Orchard’s Annual Report on Form 10-K for the year ended December 31, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchard’s views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.
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