--- title: "Ayrmid 报告了 Motixafortide 在镰状细胞病和β地中海贫血中用于干细胞动员的额外新真实世界数据" type: "News" locale: "zh-CN" url: "https://longbridge.com/zh-CN/news/274811243.md" description: "Ayrmid Ltd. 报告了关于 motixafortide 在镰状细胞病和 β-地中海贫血患者中动员造血干细胞（HSCs）的有希望的真实世界数据。研究显示，73% 的患者收集到了足够的 HSCs 以进行基因治疗，其中五名患者成功接受了治疗。作为 CXCR4 抑制剂的 motixafortide 在之前未能成功使用标准动员方法的患者中显示出了有效性。这些发现已在 2025 年 TANDEM 会议上发布，突显了 motixafortide 在增强这些疾病基因治疗可及性方面的潜力" datetime: "2026-02-04T12:16:28.000Z" locales: - [zh-CN](https://longbridge.com/zh-CN/news/274811243.md) - [en](https://longbridge.com/en/news/274811243.md) - [zh-HK](https://longbridge.com/zh-HK/news/274811243.md) --- # Ayrmid 报告了 Motixafortide 在镰状细胞病和β地中海贫血中用于干细胞动员的额外新真实世界数据 _**Key Highlights**_ - Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies - 73% (11 of 15) of patients were able to collect sufficient HSC to enable progress to gene therapy manufacturing with 5 patients receiving their gene therapy and appropriately engrafted - 73% (11 of 15) of patients had previously failed to collect sufficient cells with plerixafor - Results support the use of motixafortide as an effective single-agent mobilizer of HSC for people with sickle cell disease and in combination with G-CSF for beta-thalassemia - Findings presented at TANDEM 2026 **DUBLIN, IE / ACCESS Newswire / February 4, 2026 /** Ayrmid, Ltd. ("Ayrmid" or the "Company"), the parent company of Gamida Cell Inc., today announced further 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 Transplantation & Cellular Therapy meetings of ASTCT® and CIBMTR® (TANDEM), taking place from February 4-7, 2026 in Salt Lake City, UT. 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 15 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. Motixafortide effectively mobilized 11 patients who had previously failed plerixafor, 3 patients mobilized effectively with motixafortide used as first line mobilization therapy. In 11 patients, sufficient cells were achieved to manufacture gene therapy for both sickle cell disease and beta-thalassemia. 5 patients have received their gene therapy and have appropriately engrafted, manufacturing is underway in a further 5 patients. Patients received Casgevy and Lyfgenia, and manufacturing is underway for Casgevy, Lyfgenia and Zynteglo. This data supports that motixafortide mobilized cells can be used across different gene therapy manufacturing platforms. **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 Beta-Thalassemia** Beta-thalassemia is a genetic blood disorder characterized by reduced or absent production of beta-globin, a component of hemoglobin, which is essential for carrying oxygen in red blood cells. This condition results in anemia, which can cause symptoms such as fatigue, weakness and pallor. **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 This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit www.rns.com. **SOURCE:** Ayrmid Pharma Ltd View the original press release on ACCESS Newswire ### 相关股票 - [XLV.US](https://longbridge.com/zh-CN/quote/XLV.US.md) - [XBI.US](https://longbridge.com/zh-CN/quote/XBI.US.md) - [FBT.US](https://longbridge.com/zh-CN/quote/FBT.US.md) - [PBE.US](https://longbridge.com/zh-CN/quote/PBE.US.md) - [BLUE.US](https://longbridge.com/zh-CN/quote/BLUE.US.md) - [BLRX.US](https://longbridge.com/zh-CN/quote/BLRX.US.md) - [IBBQ.US](https://longbridge.com/zh-CN/quote/IBBQ.US.md) - [BIB.US](https://longbridge.com/zh-CN/quote/BIB.US.md) - [SBIO.US](https://longbridge.com/zh-CN/quote/SBIO.US.md) - [VHT.US](https://longbridge.com/zh-CN/quote/VHT.US.md) - [IBB.US](https://longbridge.com/zh-CN/quote/IBB.US.md) - [BBH.US](https://longbridge.com/zh-CN/quote/BBH.US.md) - [ARKG.US](https://longbridge.com/zh-CN/quote/ARKG.US.md) - [LABD.US](https://longbridge.com/zh-CN/quote/LABD.US.md) - [IHE.US](https://longbridge.com/zh-CN/quote/IHE.US.md) ## 相关资讯与研究 - [“搞钱”，才是 Revolution 千亿神话密码](https://longbridge.com/zh-CN/news/286888792.md) - [百时美施贵宝向 3 万名员工全面开放 Anthropic 公司 Claude](https://longbridge.com/zh-CN/news/287085684.md) - [定义 “医院 AI 操作系统”：北协和、南湘雅等顶级医院 为何集体选择医渡科技？](https://longbridge.com/zh-CN/news/286870257.md) - [Scholar Rock 将出席杰富瑞全球医疗保健大会，聚焦 SMA 新药 apitegromab 关键审批](https://longbridge.com/zh-CN/news/287069556.md) - [瑞银上调爵士制药评级：核心业务韧性强劲，新药 Ziihera 成增长引擎](https://longbridge.com/zh-CN/news/286920691.md)