Orchard Therapeutics to Present at ASGCT 2024

Orchard Therapeutics, now under the ownership of Kyowa Kirin, is making significant strides in the field of gene therapy. The company recently announced their participation in the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), held from May 7-11, 2024, in Baltimore. Eight presentations, including four oral and four poster sessions, will highlight advancements in their hematopoietic stem cell (HSC) gene therapy platform.

The presentations will focus on safety and efficacy data, particularly on atidarsagene autotemcel (formerly known as OTL-200, marketed as Lenmeldy™ in the U.S. and Libmeldy® in Europe). This therapy has shown promise in treating metachromatic leukodystrophy (MLD). Additionally, three presentations will address neurological, skeletal, and other outcomes from a proof-of-concept study of OTL-203 for treating the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). Another oral presentation will discuss the potential of OTL-104, an investigational therapy for a severe form of Crohn’s disease.

Leslie Meltzer, Ph.D., Chief Medical Officer of Orchard Therapeutics, emphasized the importance of these findings. "Our presentations at ASGCT add to the compendium of evidence supporting the transformative impact and broad applicability of our approach," she said. Meltzer highlighted the significance of the Presidential Symposium, which will showcase long-term follow-up data in MLD, and the invited oral presentation, which will spotlight Orchard's early pre-clinical pipeline.

The oral presentations are scheduled as follows:
- "Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal Cross-correction Mechanisms" presented by Maria Ester Bernardo on May 7.
- "Atidarsagene autotemcel (Hematopoietic Stem Cell Gene Therapy) Preserves Cognitive and Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-up" presented by Alessandro Aiuti on May 8.
- "Restoring Macrophage Immune Functions by Transplantation of Gene-modified HSCs: a Therapeutic Approach to NOD2 Crohn’s Disease" presented by Pervinder Sagoo on May 9.
- "Somatic Mutation Tracking in Hematopoietic Stem Cell Gene Therapy Reveals Absence of Clonal Hematopoiesis" presented by Francesco Gazzo on May 11.

The poster presentations include:
- "Development of an Ex Vivo Hematopoietic Stem Cell Gene Therapy for Frontotemporal Dementia (FTD)" by Yuri Ciervo on May 9.
- "Lentiviral Hematopoietic Stem Cell Gene Therapy for Late Juvenile Metachromatic Leukodystrophy" by Valeria Calbi on May 10.
- "Non-neurological, Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene Therapy in Hurler Patients: Retrospective Comparison with Allogeneic Hematopoietic Stem Cell Transplantation" by Maria Ester Bernardo on May 10.
- "Interim Analysis on Neurological Outcomes in Hurler Syndrome Patients Treated with Autologous Ex Vivo Hematopoietic Stem Cell Gene Therapy" by Maria Ester Bernardo on May 10.

In conjunction with the ASGCT presentations, Orchard's collaborators at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, published a detailed analysis in Science Translational Medicine. This study focused on early skeletal outcomes in children with MPS-IH treated with OTL-203, showing improved clinical, functional, and radiological results with a median follow-up of nearly four years.

Lenmeldy (atidarsagene autotemcel) is approved for treating children with certain forms of MLD. In Europe, it is marketed as Libmeldy. The therapy was developed in collaboration with SR-Tiget in Milan.

Orchard Therapeutics, along with Kyowa Kirin, continues to advance its mission to develop groundbreaking gene therapies. Their work is aimed at treating severe genetic disorders through HSC gene therapy, which involves genetically modifying a patient’s own blood stem cells to correct disease-causing defects.

Overall, the presentations at ASGCT and the ongoing research underscore Orchard Therapeutics' commitment to pioneering treatments that address significant medical needs in the realm of rare and severe genetic diseases.