Key Highlights
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Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies
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90% (9 of 10) of patients were able to collect sufficient HSC to enable progress to gene therapy manufacturing
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86% (6 of 7) of patients had previously failed to collect sufficient cells with plerixafor
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Results support the use of motixafortide as an effective single-agent mobilizer of HSC for people with sickle cell disease for gene therapies
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Findings presented at ASH 2025
LONDON, UK / ACCESS Newswire / December 6, 2025 / Ayrmid, Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., today announced encouraging real-world data on the use of motixafortide, a long-acting CXCR4 inhibitor licensed by Ayrmid under the brand name APHEXDA®, for mobilizing HSCs in patients with sickle cell disease undergoing gene therapy. The results were presented at the 2025 American Society of Hematology (ASH) Annual Meeting, taking place from Dec 6-9 in Orlando FL.
Motixafortide is currently FDA approved in combination with filgrastim (G-CSF) for stem cell mobilization in multiple myeloma. The product’s effectiveness and pharmacologic profile have prompted growing interest in its potential use for sickle cell disease, where collecting adequate stem cells remains a significant barrier to gene therapy. Many patients do not mobilize enough cells with standard approaches, limiting access to curative treatment.
Researchers from five treatment centers evaluated the real-world use of motixafortide in ten patients aged 14-50. Each patient underwent one or more collection cycles over two or more days to obtain the number of stem cells required for gene therapy manufacturing.
Among the seven patients who previously failed plerixafor, motixafortide led to a substantial improvement in stem cell mobilization, enabling six of the seven to successfully collect enough cells to progress to gene therapy manufacturing. Manufacturing or infusion of a gene therapy product is underway, with two patients already transplanted and demonstrating appropriate engraftment.
Dr. John Manis, Director Transfusion Medicine Service, Associate Professor of Pathology, Harvard Medical School, commented: “The approval of gene therapies for sickle cell disease has opened the door to transformative outcomes, but inadequate stem cell collection has significantly delayed manufacturing and prevented some patients from starting treatment. These findings suggest that motixafortide may help overcome this challenge, and with more studies, expand access to potentially life-changing therapies.”
About Sickle Cell Disease
Sickle cell disease, also known as sickle cell anemia, is a severe inherited blood disorder caused by a genetic mutation that leads to misshapen, rigid red blood cells. These cells can obstruct blood flow, causing sudden episodes of severe pain, known as pain crises, and leading to life-threatening complications. Ayrmid is dedicated to advancing therapies that help individuals living with sickle cell disease manage their symptoms and improve their quality of life.
About Ayrmid Ltd. and Gamida Cell
Ayrmid Ltd. is the parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed to turn cells into powerful therapeutics. Gamida Cell Inc. currently has two FDA approved products on the market in the US, namely Omisirge (please see the current full Prescribing Information, including boxed warning, here) and APHEXDA® (please see the current full Prescribing Information here). Gamida Cell operates as a wholly owned subsidiary of Ayrmid Limited, a UK entity. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, X, Facebook or Instagram.
Contacts: Media, Investors / Business Development: bd@ayrmid.com
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SOURCE: Ayrmid Pharma Ltd.
View the original press release on ACCESS Newswire
