IMAGINE is a two-part trial to evaluate the safety and preliminary efficacy of imetelstat in combination with azacitidine with or without venetoclax in patients with relapsed or refractory AML. The trial will consist of a safety run-in phase (Part A) employing a 3+3 design to monitor dose-limiting toxicities of imetelstat when administered in combination with a fixed dose of azacitidine. Part B will consist of a phase 1b trial employing a BOIN12 design to determine the optimal biological dose of imetelstat, starting at a lower dose level, in combination with azacitidine and venetoclax. Total of up to 36 participants will be accrued over 54 months at Mount Sinai Hospital. Estimated duration of trial is 114 months including recruitment, screening, treatment, and follow-up.

开始日期2026-01-01

申办/合作机构

Icahn School of Medicine at Mount Sinai

[+1]

NCT06247787

/ Recruiting临床1期IIT

A Phase 1 Study of GRN163L (Imetelstat) in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia That is in Second or Greater Relapse or That is Refractory to Relapse Therapy; Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia in First or Greater Relapse or is Refractory to Relapse Therapy

This phase I trial tests the safety, side effects, and best dose of imetelstat in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) that has not responded to previous treatment (refractory) or that has come back after a period of improvement (recurrent). Imetelstat may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Chemotherapy drugs, such as fludarabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving imetelstat in combination with fludarabine and cytarabine may work better in treating patients with refractory or recurrent AML, MDS, and JMML.

开始日期2025-02-04

申办/合作机构

The Children's Oncology Group Foundation, Inc.

[+2]

ACTRN12625000226404

/ Active, not recruiting临床2期IIT

AMLM27/ IMpress_001: A phase II study evaluating the efficacy and safety of Imetelstat in Patients with HR myelodysplastic Syndromes (MDS) or AML failing HMA-based therapy.

开始日期2023-08-07

申办/合作机构

Australasian Leukaemia & Lymphoma Group

[+1]

100 项与伊美司他相关的临床结果

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100 项与伊美司他相关的转化医学

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100 项与伊美司他相关的专利（医药）

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138

项与伊美司他相关的文献（医药）

2026-02-01·BRITISH JOURNAL OF HAEMATOLOGY

Clinical outcomes and safety in patients with lower‐risk myelodysplastic syndromes treated with imetelstat: Substudy of the phase 3 IMerge trial

Article

作者: Madanat, Yazan F. ; de Paz, Raquel ; Zeidan, Amer M. ; Kaźmierczak, Maciej ; Riggs, Jennifer ; Oliva, Esther Natalie ; Platzbecker, Uwe ; Shah, Sheetal ; Sun, Libo ; Savona, Michael R. ; Santini, Valeria ; Van Eygen, Koen ; Díez‐Campelo, María ; Santillana, Guillermo Sanz ; Komrokji, Rami S. ; Thépot, Sylvain ; Lennox, Ashley L. ; Fenaux, Pierre ; Sekeres, Mikkael A. ; Berry, Tymara ; Xia, Qi

2026-02-01·LEUKEMIA

Modulation of the clonal burden in patients with lower-risk myelodysplastic neoplasms treated with imetelstat

Article

作者: Dougherty, Souria ; Sekeres, Mikkael A ; Savona, Michael R ; Van Eygen, Koen ; Huang, Fei ; Thépot, Sylvain ; Fenaux, Pierre ; Wan, Ying ; Santini, Valeria ; Madanat, Yazan F ; Feller, Faye ; Platzbecker, Uwe ; Sun, Libo ; Raddi, Marco G ; Komrokji, Rami S ; Boyer, Thomas ; Navada, Shyamala ; Germing, Ulrich ; Berry, Tymara ; Zeidan, Amer M ; Raza, Azra ; Xia, Qi

Abstract:

Existing treatments for lower-risk myelodysplastic syndromes/neoplasms (LR-MDS) focus on symptom relief. Until recently, altering the disease course was rarely considered a therapeutic objective. The first-in-class, direct, competitive telomerase inhibitor, imetelstat, demonstrated significantly higher rates of red blood cell (RBC) transfusion independence (TI) versus placebo in patients with non-del(5q), RBC transfusion-dependent LR-MDS who were relapsed/refractory to or ineligible for erythropoiesis-stimulating agents in the Phase 3 IMerge study (NCT02598661). In this exploratory analysis of IMerge, patients treated with imetelstat had greater sustained reductions in variant allele frequency of multiple mutations versus placebo recipients, which was positively associated with RBC-TI duration. Subsequent analyses showed that 70% of patients with a cytogenetic response with imetelstat achieved ≥1-year RBC-TI. Additionally, higher rates of ≥1-year RBC-TI were observed in patients with maximum variant allele frequency reduction of ≥50% in SF3B1 (58% vs. 7%), TET2 (90% vs. 9%), DNMT3A (100% vs. 13%), or ASXL1 (50% vs. 0%) and patients with ≥50% bone marrow ring sideroblast reduction (46% vs. 0%) versus patients who did not. Lastly, 60% of patients with ≥1-year RBC-TI had ≥50% reduction in telomerase activity/human telomerase reverse transcriptase RNA. These results suggest that imetelstat targets clonal progenitor cells and may modify LR-MDS biology.

2025-12-10·JOURNAL OF CLINICAL ONCOLOGY

US Food and Drug Administration Approval Summary: Imetelstat for Selected Patients With Low- to Intermediate-1 Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia

Article

作者: Blanco, Javier G. ; Ehrlich, Lori A. ; Pulte, E. Dianne ; Bhatnagar, Vishal ; Booth, Brian ; Liu, Jiang ; Vallejo, Jonathon ; Green, Francis ; Norsworthy, Kelly J. ; De Claro, R. Angelo ; Pazdur, Richard ; Choe, Moran ; Theoret, Marc ; Wang, Yun ; Ionan, Alexei C. ; Dorff, Sarah E. ; Gehrke, Brenda ; Haupert, Spencer ; Zheng, Nan ; Kim, Nina

On June 6, 2024, the US Food and Drug Administration (FDA) approved imetelstat (RYTELO, Geron) for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring ≥4 red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents. The approval was based on a randomized (2:1), double-blind, placebo-controlled multicenter trial, Study MDS3001. In the protocol-specified primary analysis, the ≥8-week RBC transfusion independence (RBC-TI) rate was 39.8% (95% CI, 30.9 to 49.3) in the imetelstat group versus 15% (95% CI, 7.1 to 26.6) in the placebo group ( P < .001). This was supported by the rate of ≥24-week RBC-TI of 28% (95% CI, 20.1 to 37) in the imetelstat group versus 3.3% (95% CI, 0.4 to 11.5) in the placebo group ( P < .001). However, there was no major difference between arms regarding the key secondary end point of erythroid response (HI-E) per International Working Group 2006 criteria or secondary end points reflective of a disease-modifying effect such as complete remission rate and overall survival. The most common adverse reactions were thrombocytopenia, leukopenia, neutropenia, increased liver enzymes, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19, and headache. The rate of grade 3 to 4 neutropenia and thrombocytopenia were 72% and 65%, respectively, in the imetelstat arm compared with 7% and 8% in the placebo arm. Despite the high incidence of neutropenia and thrombocytopenia, the FDA determined that the benefits outweighed the risks in this patient population with high unmet need. Postmarketing requirements were issued to evaluate long-term safety and to conduct a randomized trial comparing at least two dosages of imetelstat to potentially minimize risks of imetelstat treatment and improve tolerability.

232

项与伊美司他相关的新闻（医药）

2026-03-31

·格隆汇

洞察反义寡核苷酸（ASO）药物和siRNA药物市场增长趋势：2032年规模将达13680百万美元

据QYR最新调研，2025年中国反义寡核苷酸（ASO）药物和siRNA药物市场销售收入达到了百万美元，预计2032年可以达到 13680百万美元，2026-2032期间年复合增长率为23.0%。本研究项目旨在梳理反义寡核苷酸（ASO）药物和siRNA药物领域产品系列，洞悉行业特点、市场存量空间及增量空间，并结合市场发展前景判断反义寡核苷酸（ASO）药物和siRNA药物领域内各类竞争者所处地位。小核酸药物长度较短，碱基长度少于30nt，主要通过碱基互补配对的原理作用于细胞内的mRNA，调控蛋白质表达，从而达到治疗作用。小核酸药物又可分为单链反义寡核苷酸ASO，以及双链核苷酸siRNA。反义寡核苷酸（ASO）药物与siRNA药物市场驱动因素主要包括以下：一、技术突破：从实验室到临床的跨越ASO药物的技术演进化学修饰与递送系统优化：第一代ASO药物因稳定性差、免疫原性高受限，第三代ASO通过硫代磷酸骨架修饰、糖基化改造等技术，显著提升了对核酸酶的抗性和药代动力学性能。例如，Ionis公司的Eplontersen采用配体偶联技术，实现每月一次皮下给药，患者依从性大幅提高。适应症拓展：ASO药物从遗传病（如杜氏肌营养不良症）向肿瘤、代谢疾病延伸。Geron公司的Imetelstat作为首个端粒酶抑制剂获批，为骨髓增生异常综合征提供新疗法。siRNA药物的技术革命递送系统突破：2014年GalNAc配体技术问世，解决肝脏靶向难题，推动Alnylam的Onpattro（2018年获批）成为首个siRNA药物。当前研究聚焦多器官递送，如Alnylam的mivelsiran（脑淀粉样血管病Ⅱ期）和Arrowhead的ARO-RAGE（哮喘Ⅰ期），通过脂质纳米颗粒（LNP）或抗体偶联实现跨血脑屏障靶向。长效性与可逆性优势：siRNA药物如Inclisiran（降脂）可实现半年一次给药，且作用可逆，避免了基因编辑的永久性风险。二、政策支持：全球创新药生态的催化剂监管审批加速FDA通过“快速通道”“突破性疗法”等机制加速小核酸药物审批。例如，Eplontersen从临床到获批仅用3年，远低于传统药物的10年周期。中国《“十四五”医药工业发展规划》明确支持新型递送系统研发，并优化审评流程，推动ASO/siRNA药物进入临床试验。医保覆盖与支付创新欧美医保对高价小核酸药物（如Onpattro年治疗费用约45万美元）探索“按疗效付费”模式，减轻患者负担。中国正将罕见病用药纳入医保谈判，ASO药物Spinraza（诺西那生）已通过谈判降价，可及性提升。三、市场需求：未满足临床需求的蓝海遗传病与罕见病领域ASO药物在脊髓性肌萎缩症（SMA）领域占据主导，Spinraza全球销售额超20亿美元。siRNA药物如Amvuttra（转甲状腺素蛋白淀粉样变性）填补了遗传病治疗空白。全球罕见病患者超3亿，但已获批药物仅覆盖5%，市场潜力巨大。慢性病与代谢疾病拓展siRNA药物Inclisiran将适应症从家族性高胆固醇血症扩展至动脉粥样硬化性心血管疾病，患者群体扩大10倍。代谢相关脂肪性肝炎（MASH）领域，Alnylam的Lundquist等siRNA药物进入Ⅲ期临床，预计2030年市场规模超350亿美元。四、资本投入：创新药研发的“燃料”风险投资与IPO活跃2023年全球小核酸领域发生31起交易，总金额84.96亿美元（同比+371%）。中国靖因药业完成近5000万美元B2轮融资，加速心血管siRNA管线开发。2024年，勃林格殷格翰与瑞博生物达成20亿美元合作，开发MASH的siRNA药物。跨国药企布局加速阿斯利康以35.85亿美元引进Ionis的Eplontersen，拓展心衰领域；诺华通过Leqvio（降脂siRNA）巩固心血管市场。2025年，GSK以12亿美元收购Boston Pharmaceuticals的FGF21 siRNA项目，押注代谢疾病。五、竞争格局：从“单极”到“多极”的演变国际巨头垄断与突破Ionis（ASO）和Alnylam（siRNA）占据全球主要市场份额，但Arrowhead、Dicerna等新锐通过差异化技术（如多器官递送）挑战龙头地位。礼来与Alnylam合作开发阿尔茨海默病siRNA药物，利用其递送技术突破血脑屏障。中国企业的崛起国内ASO领域：瑞博生物、悦康医药通过合作开发推进临床管线，但尚无自研产品上市。siRNA领域：圣因生物、舶望制药的多款药物进入Ⅰ期，针对高血压、高血脂等常见病，填补国内空白。六、挑战与未来趋势技术瓶颈待突破ASO药物的脱靶效应和免疫原性仍需优化；siRNA的肾脏、肺部递送效率低，限制适应症拓展。基因编辑技术（如CRISPR）的竞争压力：Verve Therapeutics的PCSK9基因编辑疗法进入临床，可能替代部分siRNA市场。中国市场受政策扶持和资本驱动，增速将高于全球平均水平，ASO/siRNA药物有望成为生物医药“新基建”的核心赛道。ASO与siRNA药物的市场爆发是技术突破、政策红利、资本催化与未满足需求的共振结果。未来，随着递送系统创新和多适应症拓展，小核酸药物将从“小众遗传病”走向“大众慢性病”，重塑全球医药格局。《2026-2032中国反义寡核苷酸（ASO）药物和siRNA药物市场现状研究分析与发展前景预测报告》研究中国市场反义寡核苷酸（ASO）药物和siRNA药物的生产、消费及进出口情况，重点关注在中国市场扮演重要角色的全球及本土反义寡核苷酸（ASO）药物和siRNA药物生产商，呈现这些厂商在中国市场的反义寡核苷酸（ASO）药物和siRNA药物销量、收入、价格、毛利率、市场份额等关键指标。此外，针对反义寡核苷酸（ASO）药物和siRNA药物产品本身的细分增长情况，如不同反义寡核苷酸（ASO）药物和siRNA药物产品类型、价格、销量、收入，不同应用反义寡核苷酸（ASO）药物和siRNA药物的市场销量等，本文也做了深入分析。历史数据为2021至2025年，预测数据为2026至2032年。反义寡核苷酸（ASO）药物和siRNA药物报告主要厂商包括：Alnylam、 Ionis、 Biogen、 Novartis、 Sarepta Therapeutics、 Therapeutics、 Akcea、 WAVElife、 ProQR、 Exicure、 Secarna、 AstraZeneca、 The WhiteOak Group, Inc.、 Dicerna、 Silence、 Arrowhead、 Miragen Therapeutics、 Genzyme、 Quark Pharmaceuticals、 Sylentis、 Avidity Biosciences、 Novo Nordisk、 Sirnaomics反义寡核苷酸（ASO）药物和siRNA药物报告主要研究产品类型包括：反义寡核苷酸（ASO）药物、 siRNA药物反义寡核苷酸（ASO）药物和siRNA药物报告主要研究应用领域，主要包括：单基因罕见病、蛋白沉积疾病、肝脏慢性疾病、其他需要查看完整版报告样本可点击链接：https://www.tydatainfo.com/reports/8741939/antisense-oligonucleotides--aso--drug-and-sirna-drug报告目录：1 反义寡核苷酸（ASO）药物和siRNA药物市场概述1.1 反义寡核苷酸（ASO）药物和siRNA药物市场概述1.2 不同产品类型反义寡核苷酸（ASO）药物和siRNA药物分析1.2.1 中国市场不同产品类型反义寡核苷酸（ASO）药物和siRNA药物规模对比（2021 VS 2025 VS 2032）1.2.2 反义寡核苷酸（ASO）药物1.2.3 siRNA药物1.3 从不同应用，反义寡核苷酸（ASO）药物和siRNA药物主要包括如下几个方面1.3.1 中国市场不同应用反义寡核苷酸（ASO）药物和siRNA药物规模对比（2021 VS 2025 VS 2032）1.3.2 单基因罕见病1.3.3 蛋白沉积疾病1.3.4 肝脏慢性疾病1.3.5 其他1.4 中国反义寡核苷酸（ASO）药物和siRNA药物市场规模现状及未来趋势（2021-2032）2 中国市场主要企业分析2.1 中国市场主要企业反义寡核苷酸（ASO）药物和siRNA药物规模及市场份额2.2 中国市场主要企业总部及主要市场区域2.3 中国市场主要厂商进入反义寡核苷酸（ASO）药物和siRNA药物行业时间点2.4 中国市场主要厂商反义寡核苷酸（ASO）药物和siRNA药物产品类型及应用2.5 反义寡核苷酸（ASO）药物和siRNA药物行业集中度、竞争程度分析2.5.1 反义寡核苷酸（ASO）药物和siRNA药物行业集中度分析：2025年中国市场Top 5厂商市场份额2.5.2 中国市场反义寡核苷酸（ASO）药物和siRNA药物第一梯队、第二梯队和第三梯队厂商及市场份额2.6 新增投资及市场并购活动3 主要企业简介3.1 Alnylam3.1.1 Alnylam公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.1.2 Alnylam 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.1.3 Alnylam在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.1.4 Alnylam公司简介及主要业务3.2 Ionis3.2.1 Ionis公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.2.2 Ionis 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.2.3 Ionis在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.2.4 Ionis公司简介及主要业务3.3 Biogen3.3.1 Biogen公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.3.2 Biogen 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.3.3 Biogen在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.3.4 Biogen公司简介及主要业务3.4 Novartis3.4.1 Novartis公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.4.2 Novartis 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.4.3 Novartis在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.4.4 Novartis公司简介及主要业务3.5 Sarepta Therapeutics3.5.1 Sarepta Therapeutics公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.5.2 Sarepta Therapeutics 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.5.3 Sarepta Therapeutics在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.5.4 Sarepta Therapeutics公司简介及主要业务3.6 Therapeutics3.6.1 Therapeutics公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.6.2 Therapeutics 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.6.3 Therapeutics在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.6.4 Therapeutics公司简介及主要业务3.7 Akcea3.7.1 Akcea公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.7.2 Akcea 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.7.3 Akcea在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.7.4 Akcea公司简介及主要业务3.8 WAVElife3.8.1 WAVElife公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.8.2 WAVElife 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.8.3 WAVElife在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.8.4 WAVElife公司简介及主要业务3.9 ProQR3.9.1 ProQR公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.9.2 ProQR 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.9.3 ProQR在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.9.4 ProQR公司简介及主要业务3.10 Exicure3.10.1 Exicure公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.10.2 Exicure 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.10.3 Exicure在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.10.4 Exicure公司简介及主要业务3.11 Secarna3.11.1 Secarna公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.11.2 Secarna 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.11.3 Secarna在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.11.4 Secarna公司简介及主要业务3.12 AstraZeneca3.12.1 AstraZeneca公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.12.2 AstraZeneca 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.12.3 AstraZeneca在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.12.4 AstraZeneca公司简介及主要业务3.13 The WhiteOak Group, Inc.3.13.1 The WhiteOak Group, Inc.公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.13.2 The WhiteOak Group, Inc. 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.13.3 The WhiteOak Group, Inc.在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.13.4 The WhiteOak Group, Inc.公司简介及主要业务3.14 Dicerna3.14.1 Dicerna公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.14.2 Dicerna 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.14.3 Dicerna在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.14.4 Dicerna公司简介及主要业务3.15 Silence3.15.1 Silence公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.15.2 Silence 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.15.3 Silence在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.15.4 Silence公司简介及主要业务3.16 Arrowhead3.16.1 Arrowhead公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.16.2 Arrowhead 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.16.3 Arrowhead在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.16.4 Arrowhead公司简介及主要业务3.17 Miragen Therapeutics3.17.1 Miragen Therapeutics公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.17.2 Miragen Therapeutics 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.17.3 Miragen Therapeutics在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.17.4 Miragen Therapeutics公司简介及主要业务3.18 Genzyme3.18.1 Genzyme公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.18.2 Genzyme 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.18.3 Genzyme在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.18.4 Genzyme公司简介及主要业务3.19 Quark Pharmaceuticals3.19.1 Quark Pharmaceuticals公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.19.2 Quark Pharmaceuticals 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.19.3 Quark Pharmaceuticals在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.19.4 Quark Pharmaceuticals公司简介及主要业务3.20 Sylentis3.20.1 Sylentis公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.20.2 Sylentis 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.20.3 Sylentis在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.20.4 Sylentis公司简介及主要业务3.21 Avidity Biosciences3.21.1 Avidity Biosciences公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.21.2 Avidity Biosciences 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.21.3 Avidity Biosciences在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.21.4 Avidity Biosciences公司简介及主要业务3.22 Novo Nordisk3.22.1 Novo Nordisk公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.22.2 Novo Nordisk 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.22.3 Novo Nordisk在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.22.4 Novo Nordisk公司简介及主要业务3.23 Sirnaomics3.23.1 Sirnaomics公司信息、总部、反义寡核苷酸（ASO）药物和siRNA药物市场地位以及主要的竞争对手3.23.2 Sirnaomics 反义寡核苷酸（ASO）药物和siRNA药物产品及服务介绍3.23.3 Sirnaomics在中国市场反义寡核苷酸（ASO）药物和siRNA药物收入（万元）及毛利率（2021-2026）3.23.4 Sirnaomics公司简介及主要业务4 中国不同产品类型反义寡核苷酸（ASO）药物和siRNA药物规模及预测4.1 中国不同产品类型反义寡核苷酸（ASO）药物和siRNA药物规模及市场份额（2021-2026）4.2 中国不同产品类型反义寡核苷酸（ASO）药物和siRNA药物规模预测（2027-2032）5 不同应用分析5.1 中国不同应用反义寡核苷酸（ASO）药物和siRNA药物规模及市场份额（2021-2026）5.2 中国不同应用反义寡核苷酸（ASO）药物和siRNA药物规模预测（2027-2032）6 行业发展机遇和风险分析6.1 反义寡核苷酸（ASO）药物和siRNA药物行业发展机遇及主要驱动因素6.2 反义寡核苷酸（ASO）药物和siRNA药物行业发展面临的风险6.3 反义寡核苷酸（ASO）药物和siRNA药物行业政策分析6.4 反义寡核苷酸（ASO）药物和siRNA药物中国企业SWOT分析7 行业供应链分析7.1 反义寡核苷酸（ASO）药物和siRNA药物行业产业链简介7.1.1 反义寡核苷酸（ASO）药物和siRNA药物行业供应链分析7.1.2 主要原材料及供应情况7.1.3 反义寡核苷酸（ASO）药物和siRNA药物行业主要下游客户7.2 反义寡核苷酸（ASO）药物和siRNA药物行业采购模式7.3 反义寡核苷酸（ASO）药物和siRNA药物行业开发/生产模式7.4 反义寡核苷酸（ASO）药物和siRNA药物行业销售模式8 研究结果9 研究方法与数据来源9.1 研究方法9.2 数据来源9.2.1 二手信息来源9.2.2 一手信息来源9.3 数据交互验证9.4 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siRNA寡核苷酸临床申请突破性疗法临床1期

2026-03-26

·GlobeNewswire-Health Care

Geron Announces Appointment of New Members to its Board of Directors

Patricia S. Andrews and Constantine Chinoporos are proven industry veterans with decades of experience leading and advising biopharmaceutical companiesFOSTER CITY, Calif., March 26, 2026 (GLOBE NEWSWIRE) -- Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today announced the appointment of Patricia S. Andrews and Constantine Chinoporos to its Board of Directors. “We are pleased to welcome Constantine and Pat to the Board, as they bring deep operational expertise cultivated over decades of biopharmaceutical industry leadership, particularly with commercial-stage organizations,” said Elizabeth O’Farrell, Chair of the Board. “Their invaluable insights will be instrumental as we execute on our strategic priorities and drive commercial growth for RYTELO®.” “I am honored to be joining the Board at such a transformational time for Geron,” said Ms. Andrews. “RYTELO is a differentiated treatment option for lower-risk myelodysplastic syndromes/neoplasms (LR-MDS) and has the potential to make a positive difference for people living with this cancer. I look forward to helping the Geron team execute on its refocused commercial strategy for RYTELO and deliver long-term growth.” “With a capable leadership team in place, a focused commercial strategy and strong financial discipline, Geron is well positioned to deliver RYTELO to eligible LR-MDS patients and create long-term value,” said Mr. Chinoporos. “I look forward to supporting the team as we work toward building Geron into a leading, sustainable hematology company.” Ms. Andrews currently serves as a member of the Board of Directors (BOD) of Glenmark Pharmaceuticals Limited, and as a director of its wholly-owned subsidiary, Ichnos Glenmark Innovation (IGI), positions she has held since August 2025. Since 2024, she has also served as a member of the BOD of Oncolytics Biotech Inc. Previously, she served as a member of the BOD and the Audit Committee of GlycoMimetics, Inc., from 2017 until its merger with Crescent Biopharma in 2025. Ms. Andrews served as Chief Executive Officer (CEO) of Sumitomo Pharma Oncology, Inc. (formerly Boston Biomedical, Inc.), and as an Executive Officer of its parent company, Sumitomo Pharma Co., Ltd., from 2017 to 2023. Ms. Andrews was also the Global Head of Oncology for Sumitomo Pharma Co., from 2020 to 2023. Ms. Andrews joined Boston Biomedical in 2013 as Executive Vice President (EVP) and Chief Commercial Officer (CCO) and was promoted to Chief Operating Officer (COO) in 2015. From 2008 to 2012, Ms. Andrews served as EVP and CCO at Incyte Corporation. Prior to Incyte, Ms. Andrews held roles of increasing responsibility at Pfizer Inc. from 1991 to 2008, including as Vice President and General Manager of the U.S. Oncology Business Unit and Vice President, Specialty Markets. Ms. Andrews holds an M.B.A. with a focus on Finance from the University of Michigan (Ann Arbor) and a B.A. in History and Political Science from Brown University. Mr. Chinoporos served as a member of the Board of Directors at Elektrofi, Inc. from 2019 until its acquisition by Halozyme Therapeutics, Inc. in 2025 and was Chairperson of the Compensation Committee. Mr. Chinoporos was COO and Chief Business Officer (CBO) at Applied Therapeutics, Inc., from 2023 until its acquisition by Cycle Pharmaceuticals in 2026. Prior to his role at Applied Therapeutics, Mr. Chinoporos served as CBO at Albireo Pharmaceuticals, Inc. from 2021 until its acquisition by Ipsen S.A. in 2023. From 2015 to 2021, Mr. Chinoporos served as CBO at Boston Pharmaceuticals, Inc. Previously, he held senior positions in worldwide licensing, business development, corporate development, corporate finance and alliance management at Sanofi S.A., Genzyme Corporation, and Eli Lilly and Company. Mr. Chinoporos currently serves as a strategic advisor to Apollo Therapeutics Ltd., a role he has held since 2023. Mr. Chinoporos holds an M.B.A. from the Johnson Graduate School of Management at Cornell University and a B.A. in History from Cornell University. About GeronGeron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO® (imetelstat) is approved in the United States and the European Union for the treatment of certain adult patients with lower-risk myelodysplastic syndromes with transfusion dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis, as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit www.geron.com or follow us on LinkedIn. Use of Forward-Looking StatementsExcept for the historical information contained herein, this press release contains forward-looking statements made pursuant to the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such statements, include, without limitation, those regarding: (i) Geron’s ability to execute on its strategic priorities and drive commercial growth for RYTELO; (ii) Geron’s ability to execute on its refocused commercial strategy for RYTELO and deliver long-term growth; (iii) RYTELO’s potential to make a positive difference for people living with this cancer; (iv) Geron’s ability to deliver RYTELO to eligible LR-MDS patients and create long-term value n; and (v) and other statements that are not historical facts, constitute forward-looking statements. These forward-looking statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements. These risks and uncertainties, include, without limitation, risks and uncertainties related to: (a) whether Geron is successful in commercializing RYTELO for the treatment of certain patients with lower-risk MDS with transfusion dependent anemia and achieves market acceptance across the breadth of the eligible patient segments in RYTELO’s approved indication; (b) whether the FDA and European Commission will approve imetelstat for other indications on the timelines expected, or at all; (c) Geron’s plans to commercialize RYTELO outside of the U.S. and risks related to operating outside of the U.S.; (d) Geron’s future opportunities and plans, including the uncertainty of future revenues, expenses and other financial performance and results; (e) whether Geron overcomes potential delays and other adverse impacts that may be caused by enrollment, clinical, safety, efficacy, technical, scientific, intellectual property, manufacturing, regulatory and healthcare challenges in order to have the financial resources for and meet expected timelines and planned milestones; (f) whether regulatory authorities permit the further development of imetelstat on a timely basis, or at all, without any clinical holds; (g) whether any future safety or efficacy results of RYTELO treatment cause its benefit-risk profile to become unacceptable; (h) whether imetelstat actually demonstrates disease-modifying activity in patients and the ability to target the malignant stem and progenitor cells of the underlying disease; (i) whether Geron meets its post-marketing requirements and commitments for RYTELO; (j) whether there are failures or delays in manufacturing or supplying sufficient quantities of RYTELO (imetelstat) or other clinical trial materials that impact commercialization of RYTELO or the continuation of clinical trials; and (k) whether Geron successfully completes its restructuring plan, manages the changes in its workforce, and realizes expected operating expense savings. Additional information on the above risks and uncertainties and additional risks, uncertainties and factors that could cause actual results to differ materially from those in the forward-looking statements are contained in Geron’s filings and periodic reports filed with the Securities and Exchange Commission under the heading “Risk Factors” and elsewhere in such filings and reports, including Geron’s annual report on Form 10-K for the year ended December 31, 2025. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, and the facts and assumptions underlying the forward-looking statements may change. Except as required by law, Geron disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances. Investors and MediaDawn SchottlandtSenior Vice President, Investor Relations and Corporate Affairsdschottlandt@geron.com

高管变更临床3期上市批准并购

2026-03-18

·BioSpace

Geron Corporation Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) - March 18, 2026

FOSTER CITY, Calif., March 18, 2026 (GLOBE NEWSWIRE) -- Geron Corporation (Nasdaq: GERN), a commercial stage biopharmaceutical company, today reported that, effective March 17, 2026, it granted stock options to purchase an aggregate of 956,250 shares of common stock to six newly hired employees as an inducement material to such employees’ acceptance of employment with Geron. The stock options have an exercise price of $1.62 per share, which is equal to the closing price of Geron’s common stock on the grant date, have a ten-year term and vest over four years, with 12.5% of the shares underlying the options vesting on the six-month anniversary of commencement of employment of such employee and the remaining shares vesting over the following 42 months in equal installments of whole shares, subject to continued employment with Geron through the applicable vesting dates. The equity awards were granted by the Compensation Committee of Geron’s Board of Directors in accordance with Nasdaq Listing Rule 5635(c)(4) and are subject to the terms and conditions of Geron’s 2018 Inducement Award Plan and the form of stock option agreement under the plan. About Geron Geron is a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer. Our first-in-class telomerase inhibitor RYTELO® (imetelstat) is approved in the United States and the European Union for the treatment of certain adult patients with lower-risk myelodysplastic syndromes with transfusion dependent anemia. We are also conducting a pivotal Phase 3 clinical trial of imetelstat in JAK-inhibitor relapsed/refractory myelofibrosis, as well as studies in other hematologic malignancies. Inhibiting telomerase activity, which is increased in malignant stem and progenitor cells in the bone marrow, aims to potentially reduce proliferation and induce death of malignant cells. To learn more, visit or follow us on LinkedIn . CONTACT: Dawn Schottlandt Senior Vice President, Investor Relations and Corporate Affairs dschottlandt@geron.com

临床3期上市批准

100 项与伊美司他相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D09629	伊美司他	-	DB14909

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
输血依赖性贫血	欧盟	Geron Corp.	2025-03-07
输血依赖性贫血	冰岛	Geron Corp.	2025-03-07
输血依赖性贫血	列支敦士登	Geron Corp.	2025-03-07
输血依赖性贫血	挪威	Geron Corp.	2025-03-07
骨髓增生异常性贫血	美国	Geron Corp.	2024-06-06

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
骨髓增生异常综合征	申请上市	美国	Geron Corp.	2024-01-12
骨髓纤维化	临床3期	美国	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	阿根廷	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	澳大利亚	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	奥地利	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	比利时	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	巴西	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	保加利亚	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	哥伦比亚	Geron Corp.	2021-04-12
骨髓纤维化	临床3期	丹麦	Geron Corp.	2021-04-12

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02426086 (IMbark, ASH2025)	临床2期	骨髓纤维化	43	Imetelstat 8.9 mg/kg	鬱糧積網簾淵觸夢鏇鹹(淵觸淵壓願製鹹糧齋鬱) = 齋膚觸廠製蓋餘鑰遞鹹範顧蓋醖艱簾衊簾鑰獵 (鹽齋築膚廠選鬱構廠艱 )	积极	2025-12-06
NCT02426086 (IMbark, ASH2025)	临床2期	骨髓纤维化	43	Imetelstat 4.4 mg/kg	襯鏇築鹹鬱壓觸膚構積(廠鏇廠選製鏇夢繭遞鹹) = 憲齋膚窪選淵選網壓鬱衊壓衊鬱艱願簾襯範憲 (蓋糧簾醖膚選網淵築築 )	积极	2025-12-06
NCT02598661 (imerge, ASH2025)	临床3期	骨髓增生异常综合征	178	Imetelstat (IME) 7.1 mg/kg	艱範壓範廠獵選襯鹹選(鬱夢憲壓膚範鹹膚網膚) = 鹽網築製鏇積鹽製夢範選壓廠鬱製襯構遞鬱衊 (憲鹹鏇艱積鹽製淵積憲, 38.3 ~ NE) 更多	积极	2025-12-06
NCT02598661 (imerge, ASH2025)	临床3期	骨髓增生异常综合征	178	Placebo	艱範壓範廠獵選襯鹹選(鬱夢憲壓膚範鹹膚網膚) = 夢構鹹構艱獵範範糧願選壓廠鬱製襯構遞鬱衊 (憲鹹鏇艱積鹽製淵積憲, 37.4 ~ NE) 更多	积极	2025-12-06
NCT02598661 (IMerge, ASH2025)	临床3期	骨髓增生异常综合征	226	Imetelstat (IME) 7.1 mg/kg	鹽鹹餘鹹糧壓糧顧憲遞(鏇窪醖憲淵選顧願鹽窪) = 餘窪網製齋繭鑰網遞醖鑰蓋築壓襯夢蓋選夢憲 (廠鹹範鬱築憲繭壓壓範 ) 更多	积极	2025-12-06
NCT05583552 (IMpress, ASH2025)	临床2期	骨髓增生异常/骨髓增生性疾病	24	Imetelstat sodium	壓選簾壓構淵壓醖選衊(簾餘繭簾網膚鏇膚繭積) = 24 SAEs occurred in 19 patients from August 2024 until 8th May 2025, of which none were deemed related to imetelstat. The most common SAEs were febrile neutropenia (n=6), disease progression/transformation to AML (n=4), sepsis (n=2) and fever (n=2). Overall, 11/24 SAEs resulted in death (n=4 disease progression or AML, n=2 cardiac/cardiorespiratory arrest, n=1 febrile neutropenia, n=1 sepsis, general health alteration and death of unknown reason). 8 of the SAEs resolved without sequelae and 2 remained unresolved. In the first cohort, 30 SAEs occurred in 18 patients, with 3 deemed possibly related to imetelstat (pneumonia [n=2], febrile neutropenia [n=1]). The most common SAEs were disease progression/transformation to AML (n=9), pneumonia (n=3), febrile neutropenia (n=3), and sepsis (n=2). 10/30 SAEs resulted in death. 遞窪膚鹽獵艱襯鬱遞衊 (獵憲積鏇齋蓋糧簾淵糧 )	不佳	2025-12-06
NCT05583552 (IMpress, ASH2025)	临床2期	骨髓增生异常/骨髓增生性疾病	24	Imetelstat sodium		不佳	2025-12-06
ASCO2025	N/A	骨髓纤维化 JAK2 mutation \| calreticulin (CALR) mutation	197	Imetelstat 9.7 mg/kg	鹽顧窪醖製齋膚網淵選(願膚襯糧範築餘窪範壓) = 42% 壓積願淵鬱觸顧衊醖簾 (襯繭獵醖蓋願糧顧艱範 ) 更多	积极	2025-05-30
NCT02598661 (IMerge, ASCO2025)	临床2/3期	骨髓增生异常综合征 ESA \| luspatercept \| lenalidomide ... 更多	226	Imetelstat	齋鏇醖選夢蓋製簾簾蓋(製簾齋獵蓋夢鬱蓋鑰艱) = 蓋製顧觸壓衊鹽襯顧顧鑰夢壓製鏇艱遞窪壓壓 (窪艱醖鬱鏇糧壓選鹹淵 ) 更多	积极	2025-05-30
NCT04576156 (IMPACTMF, EHA2025)	临床3期	骨髓纤维化	-	Imetelstat 9.4 mg/kg	窪膚網網觸膚鬱膚壓餘(網壓齋壓遞艱廠膚積獵) = 製構糧鏇衊糧鹹築憲廠鹹製憲憲齋醖積簾選餘 (製膚襯蓋願範鏇鑰糧壓 ) 更多	积极	2025-05-22
NCT02598661 (IMerge, EHA2025)	临床3期	骨髓增生异常综合征 sEPO	-	Imetelstat 7.1 mg/kg	顧淵範糧願壓廠網範網(鬱積鬱襯醖餘襯衊製積) = 繭觸醖築艱艱齋獵鬱遞網願壓觸製獵鹽艱廠廠 (網餘範顧鬱餘獵淵範醖 ) 更多	积极	2025-05-14
NCT02598661 (IMerge, EHA2025)	临床3期	骨髓增生异常综合征	178	Imetelstat	餘醖鑰網獵餘壓餘築淵(簾範遞顧襯鏇襯鹹鹹醖) = 願蓋鬱獵顧製鹹鏇製鬱窪淵廠築構顧構艱願範 (觸願淵鬱繭廠憲築築壓, 24 ~ 122) 更多	积极	2025-05-14
NCT02598661 (IMerge, EHA2025)	临床3期	骨髓增生异常综合征	178	Placebo	餘醖鑰網獵餘壓餘築淵(簾範遞顧襯鏇襯鹹鹹醖) = 夢願憲繭襯壓淵繭襯築窪淵廠築構顧構艱願範 (觸願淵鬱繭廠憲築築壓, 10 ~ 194) 更多	积极	2025-05-14
NCT02598661 (IMerge, EHA2025)	临床3期	骨髓增生异常综合征	226	Imetelstat (IME)	餘醖壓繭壓齋鏇製衊鹹(網選齋選遞衊齋鑰憲蓋) = neutropenia and thrombocytopenia were the most common adverse events seen with IME in LR-MDS 網憲築獵遞衊憲壓觸餘 (廠願鹽糧鏇憲廠觸獵醖 ) 更多	积极	2025-05-14