ORTX - Orchard Therapeutics Announces Multiple Presentations at ASGCT 2024 | Benzinga
TOKYO, LONDON and BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced four oral and four poster presentations from across its hematopoietic stem cell (HSC) gene therapy platform will be featured at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) taking place May 7-11, 2024, in Baltimore.
Featured data include several accepted abstracts and an Oral Presidential Symposium supporting the safety and efficacy of atidarsagene autotemcel (formerly OTL-200 which was recently approved as Lenmeldy™ in the U.S. and is marketed as Libmeldy® in Europe), as well as three presentations detailing neurological, skeletal, and other clinical outcomes from a proof-of-concept (PoC) study of investigational OTL-203 in the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). In addition, Orchard Therapeutics will give an invited oral presentation highlighting the potential of OTL-104, a pre-clinical HSC gene therapy developed by its in-house research team, to address a severe and treatment refractory form of Crohn's disease.
"Our presentations at ASGCT add to the compendium of evidence supporting the transformative impact and broad applicability of our approach," said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics. "In particular, the Presidential Symposium highlighting the long-term follow-up data in MLD and the invited oral presentation spotlighting our early pre-clinical pipeline continue to demonstrate the scientific interest generated by our platform and underscore our commitment to leveraging insights gleaned from our late-stage portfolio to inform the development strategy and indication prioritization of our earlier-stage programs."
Details of the oral presentations are follows (all times in EDT):
- Title: Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal Cross-correction Mechanisms
Date/Time: Tuesday, May 7 at 1:30 p.m.
Presenter: Maria Ester Bernardo
- Title: Atidarsagene autotemcel (Hematopoietic Stem Cell Gene Therapy) Preserves Cognitive and Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-up (Oral Presidential Symposium)
Date/Time: Wednesday, May 8 at 11:15 a.m.
Presenter: Alessandro Aiuti
- Title: Restoring Macrophage Immune Functions by Transplantation of Gene-modified HSCs: a Therapeutic Approach to NOD2 Crohn's Disease (Invited Oral Presentation)
Date/Time: Thursday, May 9 at 9:18 a.m.
Presenter: Pervinder Sagoo
- Title: Somatic Mutation Tracking in Hematopoietic Stem Cell Gene Therapy Reveals Absence of Clonal Hematopoiesis
Date/Time: Saturday, May 11 at 11:45 a.m.
Presenter: Francesco Gazzo
Details of the poster presentations are as follows (all times in EDT):
- Title: Development of an Ex Vivo Hematopoietic Stem Cell Gene Therapy for Frontotemporal Dementia (FTD)
Date/Time: Thursday, May 9 from noon to 1:30 p.m. and from 5:30 to 7:00 p.m.
Presenter: Yuri Ciervo
Poster #1136
- Title: Lentiviral Hematopoietic Stem Cell Gene Therapy for Late Juvenile Metachromatic leukodystrophy
Date/Time: Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00 p.m.
Presenter: Valeria Calbi
Poster #1905
- Title: Non-neurological, Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene therapy in Hurler Patients: Retrospective Comparison with Allogeneic Hematopoietic Stem Cell Transplantation?
Date/Time: Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00 p.m.
Presenter: Maria Ester Bernardo
Poster #1904
- Title: Interim Analysis on Neurological Outcomes in Hurler Syndrome Patients Treated with Autologous Ex Vivo Hematopoietic Stem Cell Gene Therapy
Date/Time: Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00 p.m.
Presenter: Maria Ester Bernardo
Poster #1903
Early skeletal outcomes from OTL-203 PoC study in ...