Motixafortide alone and in combination with natalizumab can support the collection of the large number of stem cells required by gene therapies for sickle cell disease within a single apheresis cycle
Key Highlights
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Motixafortide alone and in combination with natalizumab (VLA-4 inhibitor) led to robust hematopoietic stem cell (HSC) mobilization predicting 16.9 million and 19.6 million cells/kg collected in a single apheresis session.
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Motixafortide alone and in combination with natalizumab mobilized 2.7 and 2.8 fold greater HSCs, relative to plerixafor
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Results support the use of motixafortide as an effective single-agent mobilizer of HSCs for people with sickle cell disease enabling accelerated access to gene therapies
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Data from study shows that motixafortide was safe and well tolerated
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Findings presented at ASH 2025
Ayrmid, Ltd. (“Ayrmid” or the “Company”), the parent company of Gamida Cell Inc., today announced encouraging new 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.
This first-in-human trial, conducted in collaboration with Washington University School of Medicine in St. Louis, explored alternative HSC mobilization strategies that could significantly improve the treatment journey of patients with sickle cell disease seeking gene therapy.
Dr. Zachary Crees, Principal Investigator for the trial, Division of Oncology, Washington University School of Medicine commented: “Currently available gene therapies for sickle cell disease rely on the collection of significant quantities of hematopoietic stem cells, posing challenges for many patients. The findings in this trial indicate that patients with sickle cell disease given motixafortide alone, or in combination with natalizumab, could mobilize and potentially collect the number of stem cells required for approved gene therapies in a single apheresis cycle.”
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 research 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.
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
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