Orchard Therapeutics Announces Eight Presentations Across Gene Therapy Portfolio at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting
Most Comprehensive Presentation of Orchard Clinical Data to Date; Spans Five Disease Areas and Highlights Broad Applicability of Hematopoietic Stem Cell Gene Therapy Approach
Accepted Abstracts Include Data on Investigational Gene Therapies for Neurometabolic Conditions, Including Updated Interim Data from Proof-of-Concept Trial in OTL-203 for Mucopolysaccharidosis Type I
BOSTON and LONDON,
“Data to be presented at ASGCT represent tremendous progress in our mission and continued momentum across our clinical programs,” said
Gaspar continues, “These programs are a result of tireless work by our research partners, and I want to thank them, the clinicians and patients involved in the clinical trials for showing how hematopoietic stem cell gene therapy can potentially change the lives of individuals with these devastating conditions.”
Of note, two of the eight presentations at ASGCT focus on new interim data from OTL-203, Orchard’s investigational gene therapy for the treatment of MPS-I, a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme that results in the accumulation of complex carbohydrates called glycosaminoglycans and is characterized by neurological, skeletal and cardiovascular problems.
The presentations also include the full integrated analysis of the registrational data set of OTL-101 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID), which includes all patients treated in the
The presentations are listed below and the full abstracts for the oral and poster presentations are available on the ASGCT meeting website. All times listed are Eastern Time (ET).
Invited Oral Presentation Details
Long-term Outcomes of Strimvelis (ex vivo gene therapy for ADA-SCID)
Session: The Long-term View: Extended follow up of Patients in Pivotal Gene Therapy Trials
Date and time:
HSC-based Strategies to Treat CNS Disorders: Focus Hurler/MPS1H
Session: Gene and Cell Therapeutic Strategies to Treat Disorders of the CNS – An International Perspective - Organized by the International Committee
Date and time:
Oral Presentation Details
Preliminary Outcomes of Haematopoietic Stem Cell Gene Therapy in a Patient with Mucopolysaccharidosis IIIA*
Abstract number: 525
Session: Clinical Gene Therapies for Inborn Errors of Metabolism
Date and time:
Pathophysiological Mechanisms of Bone Defects and Impact of Ex Vivo Hematopoietic Stem Cell Gene Therapy in Mucopolysaccharidosis Type I Hurler
Abstract number: 528
Session: Clinical Gene Therapies for Inborn Errors of Metabolism
Date and time:
Late Phases of Hematopoietic Reconstitution in Metachromatic Leukodystrophy Gene Therapy Patients are Characterized by Lineage Commitment of Individual HSC Clones
Abstract number: 530
Session: Clinical Gene Therapies for Inborn Errors of Metabolism
Date and time:
Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Two Year Follow-up Results
Abstract number: 1300
Session: Clinical Trials Spotlight Symposium
Date and time:
Poster Presentation Details
Clinical Outcomes from a Phase I/II Gene Therapy Trial for Patients Affected by Severe Transfusion Dependent Beta-Thalassemia: Two-year Follow Up
Abstract number: 376
Session: Hematologic and Immunologic Diseases
Date and time:
Liquid-Biopsy-Integration-Site-Sequencing for the Retrieval of Vector Integrations from Cell-Free DNA and Detection of Early Premalignant Expansions Hidden in Solid Tissues
Abstract number: 391
Session: Hematologic and Immunologic Diseases
Date and time:
*Patient was treated by the Royal Manchester Children’s Hospital (RMCH) 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 investigational gene therapy product (OTL-201) and is funding the proof-of-concept clinical trial being conducted at RMCH, which utilizes the same technology and procedures that were used to treat this first MPS-IIIA patient.
About Orchard
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Source: Orchard Therapeutics (Europe) Limited