--- title: "Ayrmid 報告了 Motixafortide 在鐮狀細胞病和β地中海貧血中用於幹細胞動員的額外新真實世界數據" type: "News" locale: "zh-HK" url: "https://longbridge.com/zh-HK/news/274811243.md" description: "Ayrmid Ltd. 報告了關於 motixafortide 在鐮狀細胞病和 β-地中海貧血患者中動員造血幹細胞（HSCs）的有希望的真實世界數據。研究顯示，73% 的患者收集到了足夠的 HSCs 以進行基因治療，其中五名患者成功接受了治療。作為 CXCR4 抑制劑的 motixafortide 在之前未能成功使用標準動員方法的患者中顯示出了有效性。這些發現已在 2025 年 TANDEM 會議上發佈，突顯了 motixafortide 在增強這些疾病基因治療可及性方面的潛力" 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 報告了 Motixafortide 在鐮狀細胞病和β地中海貧血中用於幹細胞動員的額外新真實世界數據 _**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 ### 相關股票 - [XLV.US](https://longbridge.com/zh-HK/quote/XLV.US.md) - [XBI.US](https://longbridge.com/zh-HK/quote/XBI.US.md) - [FBT.US](https://longbridge.com/zh-HK/quote/FBT.US.md) - [PBE.US](https://longbridge.com/zh-HK/quote/PBE.US.md) - [BLUE.US](https://longbridge.com/zh-HK/quote/BLUE.US.md) - [BLRX.US](https://longbridge.com/zh-HK/quote/BLRX.US.md) - [IBBQ.US](https://longbridge.com/zh-HK/quote/IBBQ.US.md) - [BIB.US](https://longbridge.com/zh-HK/quote/BIB.US.md) - [SBIO.US](https://longbridge.com/zh-HK/quote/SBIO.US.md) - [VHT.US](https://longbridge.com/zh-HK/quote/VHT.US.md) - [IBB.US](https://longbridge.com/zh-HK/quote/IBB.US.md) - [BBH.US](https://longbridge.com/zh-HK/quote/BBH.US.md) - [ARKG.US](https://longbridge.com/zh-HK/quote/ARKG.US.md) - [LABD.US](https://longbridge.com/zh-HK/quote/LABD.US.md) - [IHE.US](https://longbridge.com/zh-HK/quote/IHE.US.md) ## 相關資訊與研究 - [儲局會議記錄顯示 大多數官員警告通脹率若持續高於 2% 需考慮加息](https://longbridge.com/zh-HK/news/287171073.md) - [BioLineRx 的藥物在腦腫瘤動物模型中展現了抗腫瘤活性](https://longbridge.com/zh-HK/news/286973840.md) - [鉅亨速報 - Factset 最新調查：Roivant Sciences LtdROIV-US 的目標價調升至 38 元，幅度約 8.57%](https://longbridge.com/zh-HK/news/287126179.md) - [復宏漢霖：注射用 HLX43 用於治療晚期非小細胞肺癌的國際多中心 2 期臨牀研究完成首例患者給藥](https://longbridge.com/zh-HK/news/287195179.md) - [川普投資組合神操作？Medicare 政策翻盤前 精準大買聯合健康](https://longbridge.com/zh-HK/news/287039034.md)