The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

开始日期2022-07-13

申办/合作机构

Ionis Pharmaceuticals, Inc.

NCT05139810

/ Completed临床3期

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).

开始日期2021-12-03

申办/合作机构

Ionis Pharmaceuticals, Inc.

NCT04549922

/ Completed临床2期IIT

Antisense Therapy to Block the Kallikrein-kinin Pathway in COVID-19: A Phase II Randomized Controlled Trial

Up to 1/3 of all patients infected with COVID-19 can develop complications that require hospitalization. Severe pneumonia associated with acute respiratory distress syndrome (ARDS) is the most threatening and feared complication of COVID-19 infection, with mortality rates close to 50% in some groups.
Autopsies between these severe cases reveal severe capillary involvement, with signs of intense inflammatory changes, microvascular thrombosis, endothelial injury and abnormal tissue repair. The available evidence suggests that abnormal activation or imbalance in the counter-regulation of the kallikrein-kinin system may play a central role in a positive feedback cycle, leading to consequent diffuse microangiopathy. Blockade of the kallikrein-kinin system can therefore prevent deterioration of lung function by reducing inflammation, edema and microthrombosis.
The objective of this phase IIb study is to assess the preliminary effects on the oxygenation parameters of an antisense oligonucleotide that inhibits pre-kallikrein synthesis in patients with moderate to severe COVID-19.

开始日期2020-10-19

申办/合作机构

Associacao Beneficente Siria

[+2]

100 项与 Donidalorsen 相关的临床结果

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100 项与 Donidalorsen 相关的转化医学

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100 项与 Donidalorsen 相关的专利（医药）

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项与 Donidalorsen 相关的文献（医药）

2025-11-01·CTS-Clinical and Translational Science

Exposure–Response Analysis of Donidalorsen for the Treatment of Hereditary Angioedema

Article

作者: Witjes, Han ; Cohn, Danny M. ; Singh, Pratap ; Diep, John K. ; Bordone, Laura ; Newman, Kenneth B. ; Kleijn, Huub Jan ; Gao, Xiang

ABSTRACT:

Hereditary angioedema (HAE) is characterized by recurrent attacks of severe tissue swelling. In the OASIS‐HAE phase 3 study (NCT05139810), donidalorsen, an RNA‐targeted antisense oligonucleotide that reduces prekallikrein production in the liver, significantly reduced HAE attack rates. To characterize the relationship between prekallikrein concentrations and HAE attack rates following donidalorsen and predict the efficacy of potential dosing regimens, an exposure–response model was developed using data from OASIS‐HAE. Simulations were conducted to evaluate the following regimens: 80 mg once every 4 weeks (Q4W), 8 weeks (Q8W), 1 month (Q1M), and 2 months (Q2M), and a switch to Q2M dosing for patients who were attack‐free for 3 months on the Q1M regimen. The relationship between prekallikrein concentrations and HAE attack rates was well characterized by a sigmoidal E max (maximum effect) model with baseline attack rate and baseline prekallikrein concentration included as covariates. The average prekallikrein concentration estimated to result in a 90% reduction in attack rate (EC 10 ) was 47.1 mg/L. Predicted percent reductions in attack rates at steady state were similar for Q4W (84.1%) vs. Q1M (82.9%) and Q8W (72.6%) vs. Q2M (70.2%) dosing. Predicted reductions in attack rate remained similar and clinically meaningful in patients who switched from Q1M to Q2M dosing (94.0% in month 1 and 91.3% in month 2 of the 2‐month dosing interval at steady state). Overall, exposure–response analyses supported the efficacy of Q1M and Q2M dosing and indicated that switching to Q2M dosing could be a viable approach for patients who are attack‐free on the Q1M regimen.

2025-09-01·Journal of Allergy and Clinical Immunology-In Practice

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis

Article

作者: Lin, Tao ; Manning, Michael E ; Perego, Francesca ; Craig, Timothy ; Treadwell, Sabrina ; Newman, Kenneth B ; Lumry, William R ; Bordone, Laura ; Jacobs, Joshua S ; Yarlas, Aaron ; Riedl, Marc A ; Bernstein, Jonathan A ; Cohn, Danny M ; Banerji, Aleena ; Gierer, Selina ; Wedner, H James

BACKGROUND:

Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by episodes of tissue swelling. Donidalorsen, an investigational ligand-conjugated antisense oligonucleotide, reduces plasma prekallikrein production.

OBJECTIVE:

We report an interim analysis on safety, efficacy, quality of life (QoL), and treatment preference and satisfaction from an ongoing open-label phase 3 study (OASISplus Switch cohort, NCT05392114).

METHODS:

Patients with HAE receiving stable doses (≥12 weeks) of lanadelumab, complement protein 1 inhibitor, or berotralstat switched to donidalorsen 80 mg subcutaneously every 4 weeks, using a predefined algorithm. The primary end point was the incidence and severity of treatment-emergent adverse events. Other end points included change in HAE attack rate, angioedema-QoL score, disease control (≥10 points on the Angioedema Control Test), and treatment preference and satisfaction at week 16, compared with baseline on prior treatment.

RESULTS:

A total of 65 patients were enrolled; 32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat. At cutoff, 58 were ongoing in the study (89%). Forty-five patients (70%) reported treatment-emergent adverse events; 62% were unrelated to donidalorsen. At week 16, total HAE attack rates had decreased by 62%. Hereditary angioedema attack rates decreased by 65%, 41%, and 73%, and mean angioedema-QoL scores improved by 8.4, 9.6, and 17.1 points for patients switching from lanadelumab, C1INH, and berotralstat, respectively. More patients reported well-controlled disease (93% vs 67%), and most patients preferred donidalorsen over their prior treatment, with improved treatment satisfaction.

CONCLUSIONS:

Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred donidalorsen over their prior treatment. Further analyses are planned at week 52.

2025-08-01·ALLERGY

Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema

Article

作者: Treadwell, Sabrina ; Yarlas, Aaron ; Cohn, Danny M. ; Riedl, Marc A. ; Wang, Sophie ; Bordone, Laura ; Newman, Kenneth B.

ABSTRACT:

Background:

Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, frequently severe swelling that negatively impacts patients' quality of life (QoL). In the phase III OASIS‐HAE study (NCT05139810), donidalorsen reduced HAE attack rate, increased disease control, and improved QoL. Here, we report further analysis of donidalorsen's impact on QoL and other patient‐reported outcomes (PROs).

Methods:

This double‐blind, placebo‐controlled study randomized patients with HAE to donidalorsen 80 mg or placebo once every 4 (Q4W) or 8 weeks (Q8W) over 24 weeks. PROs included Angioedema (AE)‐QoL Questionnaire, Angioedema Control Test (AECT), Patient Global Impression of Severity (PGIS), and Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questions (WPAI+CIQ).

Results:

Ninety patients received donidalorsen Q4W (n = 45), donidalorsen Q8W (n = 23), or placebo (n = 22). A larger percentage of the donidalorsen Q4W group (88%) achieved clinically meaningful improvement (≥ 6‐point reduction) in AE‐QoL total score vs. placebo (45%). Both donidalorsen groups reported larger least‐squares mean (LSM) changes from baseline to week 24 vs. placebo in AE‐QoL functioning (difference: Q4W, −24.5; Q8W, −16.1), fears/shame (Q4W, −23.9; Q8W, −20.1), and nutrition (Q4W, −15.7; Q8W, −10.7) domains. Donidalorsen improved AECT scores vs. placebo (difference: Q4W, 6.0; Q8W, 4.1). A greater proportion of the donidalorsen Q4W group reported decreased disease severity vs. the placebo group (PGIS; 82% vs. 44%). Donidalorsen Q4W showed benefits vs. placebo in the presenteeism, overall work/school impairment, and activity impairment domains of the WPAI+CIQ.

Conclusions:

Donidalorsen significantly improved QoL and other PROs vs. placebo in patients with HAE.

137

项与 Donidalorsen 相关的新闻（医药）

2025-12-15

·Business Wire

GENESIS Pharma announces a new partnership with Otsuka Pharmaceutical Europe Ltd. for the commercialization of donidalorsen for hereditary angioedema in Central and Eastern Europe

ATHENS, Greece--(BUSINESS WIRE)-- ANNOUNCEMENT FOR EUROPEAN MEDICAL & PHARMACEUTICAL TRADE MEDIA AND EUROPEAN FINANCIAL MEDIA ONLY GENESIS Pharma, a regional biopharma company focused on the commercialization of innovative medicines in Central and Eastern Europe, announces an exclusive agreement with Otsuka Pharmaceutical Europe Ltd. (OPEL), the European operation of global healthcare company Otsuka Pharmaceutical Co., Ltd., for donidalorsen. Under the terms of the agreement, GENESIS Pharma will exclusively distribute and commercialize donidalorsen in fourteen markets: Bulgaria, Croatia, Cyprus, Czech Republic, Estonia, Greece, Hungary, Latvia, Lithuania, Malta, Poland, Romania, Slovakia and Slovenia. In November 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for donidalorsen in the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. The CHMP opinion is currently under review by the European Commission (EC), to determine donidalorsen’s authorisation in the European Union (EU). Donidalorsen is an investigational RNA-targeted medicine designed to reduce the production of prekallikrein (PKK), interrupting the pathway that leads to HAE attacks1,2. Mr. Constantinos Evripides, Managing Director of GENESIS Pharma stated: “We are excited to expand our international partnerships in the CEE region through this new collaboration with Otsuka Pharmaceutical Europe Ltd., a company focusing on some of the world’s unresolved health issues. The addition of donidalorsen to our robust rare disease medicines portfolio is another step forward in our mission to support patients with rare and serious conditions, ensuring access to innovative treatments in our region. Thus, we look forward to the forthcoming decision from the European Commission.” Donidalorsen was discovered and developed by Ionis Pharmaceuticals, Inc. (Ionis). In December 2023, Otsuka acquired exclusive rights to commercialize donidalorsen in Europe through a licensing agreement with Ionis. About Hereditary Angioedema (HAE) HAE is a rare, chronic and potentially life-threatening genetic condition that involves recurrent, unpredictable attacks of swelling episodes that can affect the extremities, face, abdomen, genitals and even the larynx, posing serious risk for patients.3 Symptoms of HAE usually begin in childhood or early adulthood, with some individuals experiencing their first episodes as young as two years old.4,5 HAE imposes a significant burden on patients and their families, with unpredictable and often debilitating attacks which can disrupt daily life, limit activities and reduce productivity at work or school. 6-8 HAE is most commonly caused by an inherited deficiency or dysfunction of C1 esterase inhibitor, which leads to uncontrolled activation of the plasma kallikrein-kinin system and excessive production of bradykinin.5,9,10 Approximately 15,000 individuals are living with HAE in Europe with a worldwide prevalence estimated to be 1 in 50,000.3,5,11 About GENESIS Pharma GENESIS Pharma is a regional biopharma company focused on the commercialization of innovative biopharmaceutical products targeting severe and rare diseases in Central and Eastern Europe. Established in 1997, GENESIS Pharma was among the first pharmaceutical companies in Europe to specialize in the marketing, sales and distribution of biopharmaceutical products. GENESIS Pharma maintains a strong portfolio in therapeutic areas with high unmet medical need through long standing strategic alliances with some of the leading global biopharma companies. For more information, please visit www.genesispharma.com and follow us on LinkedIn. REFERENCES Riedl MA, et al. N Engl J Med 2024;391(1):21–31. Riedl MA, et al. J Allergy Clin Immunol Pract 2025;13(9):2381–2389. Raasch J, et al. World Allergy Organ J 2023;16(6):100792. Busse P, et al. J Allergy Clin Immunol Pract 2021;9(1):132–150.e3. Maurer M, et al. Allergy 2022;77(7):1961–1990. Aygoren-Pursun E, et al. Orphanet J Rare Dis 2014;9:99. Chong-Neto HJ, World Allergy Organ J 2023;16(3):100758. Mendivil J, et al. Orphanet J Rare Dis 2021;16:94. Santacroce R, et al. Review J Clin Med 2021:10(9):2023. Longhurst HJ, Bork K. Br J Hosp Med (Lond) 2019:2;80(7):391–398. Lumry WR. Hereditary angioedema: the economics of treatment of an orphan disease. Front Med (Lausanne). 2018;5:22.

引进/卖出

2025-12-10

·药物发现笔记

2025年FDA新药批准总结

截至11月底，2025年已有39款新药获批，预计全年总数将少于往年平均50种。本期《药物发现笔记》重点介绍其中部分，包括加速批准、罕见病药物以及具有创新作用机制的首创疗法。这些新药为患者、照护者和医生提供了治疗严重疾病，以及此前治疗选择有限的疾病的新工具。约有五分之一（20%）的新药批准基于替代终点（而非长期临床结局）获得了加速批准，从而加快了患者可及性。2025年，FDA基于特定生物标志物驱动适应症，对三款治疗局部晚期或转移性非鳞状非小细胞肺癌的药物给予了加速批准： Telisotuzumab vedotin-tllv (Emrelis™)：来自艾伯维的一款抗体偶联药物，靶向c-Met蛋白过度表达的肿瘤。 Zongertinib (Hernexeos®)：来自勃林格殷格翰的一款不可逆、共价HER2选择性（对EGFR无影响）酪氨酸激酶抑制剂，用于治疗具有HER2酪氨酸激酶结构域激活突变的肿瘤。 Sevabertinib (Hyrnuo™)：来自拜耳的一款可逆性HER2/EGFR突变靶向酪氨酸激酶抑制剂，同样用于HER2突变非小细胞肺癌。这些批准凸显了精准肿瘤学的进步，为既往接受过全身治疗的特定分子亚型非小细胞肺癌患者提供了新颖的靶向疗法——涵盖生物制剂和口服小分子药物。两款针对免疫球蛋白A肾病（IgAN）的新型疗法也获得了加速批准，代表了不同的机制途径： Atrasentan (Vanrafia®)：来自诺华的一款口服选择性内皮素A（endothelin type A）受体拮抗剂。 Sibeprenlimab-szsi (Voyxact®)：来自大塚制药的一款皮下注射单克隆抗体，可阻断增殖诱导配体(A Proliferation Inducing Ligand)。两者均适用于降低有快速疾病进展风险的成年原发性IgAN患者的蛋白尿，分别通过抑制内皮素通路和调节B细胞，为减缓肾功能衰退提供了新选择。 2025年新药批准中超过半数（53%）针对罕见病，突显了制药业对特殊患者群体未满足需求的重大关注。今年，FDA批准了三款治疗遗传性血管性水肿（HAE）的新疗法，体现了多样化的治疗策略： Garadacimab-gxii (ANDEMBRY®)：来自CSL Behring的一款单克隆抗体，通过抑制因子XIIa来预防HAE发作。 Donidalorsen (DAWNZERA™)：来自Ionis Pharmaceuticals的一款靶向前激肽释放酶（prekallikrein）的反义寡核苷酸（ASO），用于预防性发作控制。 Sebetralstat (EKTERLY®)：来自KalVista Pharmaceuticals的一款口服血浆激肽释放酶（kallikrein）抑制剂，用于治疗急性发作。这些批准——涵盖单克隆抗体、RNA靶向和口服小分子药物模式——共同丰富了HAE的治疗格局，为长期预防和按需管理提供了靶向、机制各异的选项。 Mirdametinib (Gomekli™) 是SpringWorks Therapeutics开发的一款小分子激酶抑制剂，是2025年值得关注的一项罕见病批准。它靶向MEK1/2蛋白以抑制下游ERK磷酸化，从而治疗不适合手术的1型神经纤维瘤病相关丛状神经纤维瘤症状性患者（NF1-PN），为该特定遗传疾病的儿童和成人患者提供了一个靶向治疗选择。在总计39项批准中，约半数（50%）代表了首创疗法（first-in-class），突显了对新作用机制的高度关注。 Suzetrigine (Journavx™) 由Vertex Pharmaceuticals, Inc.开发，是一款首创的非阿片类镇痛药。该小分子选择性阻断NaV1.8电压门控钠通道，这是治疗中重度急性疼痛的一种新机制。 2025年的批准包括两款开创性的首创RNA干扰疗法： Fitusiran (Qfitlia™)：由Alnylam发现并开发（后被赛诺菲收购）的双链siRNA，可降解抗凝血酶（antithrombin）mRNA，预防A型或B型血友病的出血事件。这是首个用于血友病的RNAi疗法。 Plozasiran (Redemplo®)：来自Arrowhead Pharmaceuticals的GalNAc偶联siRNA，靶向并降解apoC-III mRNA以降低家族性乳糜微粒血症综合征患者的甘油三酯，标志着针对这种罕见脂质紊乱的首个且唯一一个RNAi治疗方法。这两款皮下给药疗法均展示了RNAi向新疾病领域的扩展，提供了新颖的基因沉默干预手段。 2025年，两款首创的布鲁顿酪氨酸激酶抑制剂（BTKi）被批准用于不同的自身免疫性疾病： Rilzabrutinib (Wayrilz™)：赛诺菲开发的一款共价可逆BTK抑制剂，是首个获批治疗持续性或慢性免疫性血小板减少症（ITP）的药物。 Remibrutinib (Rhapsido®)：诺华开发的一款选择性共价BTK抑制剂，是首个FDA批准用于治疗对H1抗组胺药无反应的慢性自发性荨麻疹（CSU）的BTK抑制剂。这两款口服小分子药物通过靶向BTK来应对免疫介导的病理过程，代表了一种新的治疗策略。 Brensocatib (Brinsupri™)是Insmed Incorporated的一款口服二肽基肽酶1（DPP1）抑制剂，Nerandomilast (Jascayd®)是勃林格殷格翰的一款口服磷酸二酯酶4B（PDE4B）抑制剂，两者都是针对严重进展性呼吸道疾病的首创疗法。Brensocatib是首个通过抑制中性粒细胞丝氨酸蛋白酶活化来减少非囊性纤维化支气管扩张急性加重的获批疗法；而Nerandomilast是首个获批通过靶向纤维化通路中的环磷酸腺苷（cAMP）信号来减缓特发性肺纤维化疾病进展的PDE4B抑制剂。这两款药物为既往治疗手段有限的疾病提供了新颖、机制特异的口服选择。爱尔康公司的Acoltremon (Tryptyr®)和LENZ Therapeutics, Inc.的Aceclidine (Vizz™)是两款首创的眼科小分子药物，通过新机制应对常见的年龄相关性视力障碍。Acoltremon是一种TRPM8受体激动剂，是首个此类干眼病疗法。Aceclidine是一种胆碱能毒蕈碱受体激动剂，是首个获批的基于Aceclidine的滴眼液，用于改善老花眼的近视力。两者均代表了开创性的局部治疗方法——以滴眼液形式给药——为先前使用更宽泛或特异性较低疗法的疾病提供了新的靶向药理学途径。拜耳开发的Elinzanetant (Lynkuet®)代表了女性健康领域的重大进展，它是首个也是唯一一个双重神经激肽1和神经激肽3受体拮抗剂。这款口服疗法专门获批用于治疗因绝经引起的中重度血管舒缩症状，提供了一个新颖的非激素选择。 2025年获批的针对神经病学、传染病、皮肤病学、血液学和肿瘤学等领域严重、难治性疾病的其它值得关注的首创疗法还包括：阿斯利康和第一三共的Datopotamab deruxtecan-dlnk (Datroway®)：一款靶向Trop-2的抗体偶联药物，可递送拓扑异构酶I抑制剂有效载荷，用于治疗HR+/HER2-转移性乳腺癌。葛兰素史克的Gepotidacin (Blujepa™)是一种小分子抗生素，通过抑制细菌拓扑异构酶II和IV来治疗单纯性尿路感染，代表了近30年来首个用于单纯性尿路感染的新型口服抗生素类别。强生公司的Nipocalimab-aahu (Imaavy™)：首个通过阻断FcRn来减少全身性重症肌无力患者致病性IgG的单克隆抗体。再生元制药的Linvoseltamab-gcpt (Lynozyfic™)：一款BCMAxCD3双特异性T细胞衔接器，用于治疗复发/难治性多发性骨髓瘤患者。利奥制药的Delgocitinib (Anzupgo®)：首个用于治疗中重度慢性手部湿疹的泛JAK抑制剂外用软膏。 Copyright Notice: Proper citation must include: “WeChat Public Account: 药物发现笔记 (Drug Discovery Notes) + [Article Title] + [Publication Date]”. Disclaimer: This platform is dedicated to sharing and discussing scientific research in drug discovery. Content herein does not constitute clinical treatment recommendations. The opinions expressed are solely those of the authors and do not represent the opinions of any affiliated organizations. This article is not investment advice. 引用时须注明：“微信公众号：药物发现笔记（Drug Discovery Notes）+ [文章标题] + [发表日期]”。声明：本公众号平台仅限于药物发现科学研究内容分享和讨论，不作为临床治疗方案推荐。本文仅代表个人观点，不代表公司立场，不作为投资建议。

加速审批申请上市临床申请寡核苷酸上市批准

2025-12-10

·今日头条

武汉海特生物董秘看好CGT方向参股管线获FDA临床试验批准

武汉海特生物制药股份有限公司董事会秘书杨坤近期表示，从技术演变路径看，单抗、双抗、ADC等靶向药物的商业化成功，充分证明细胞与基因治疗（CGT）方向技术上可行，未来商业价值可期，因此公司看好CGT领域。 1. 海特生物的CGT布局：从参股管线到CAR-T协同海特生物的CGT布局已落地具体项目。其参股公司中眸医疗科技（武汉）有限公司的“ZM-02眼用注射液治疗晚期视网膜色素变性”药物管线，于2025年11月获得美国FDA临床试验批准。 ZM-02是新一代光遗传学基因疗法，通过单次玻璃体腔注射将新型光敏蛋白基因递送至视网膜细胞，使缺乏功能性光感受器的患者感知光信号；与传统针对特定基因突变的疗法不同，ZM-02采用非基因突变位点依赖性策略，可覆盖遗传性及获得性视网膜退行性疾病（如年龄相关性黄斑变性）。临床前研究显示，该疗法在视网膜色素变性小鼠模型中实现稳定蛋白表达，光敏感度及视功能恢复能力卓越；正在进行的首次人体MOON临床试验（NCT06292650）显示，其可显著改善晚期视网膜色素变性患者的视力及生活质量，且安全性良好。此外，海特生物还围绕CAR-T疗法开展协同研究。2025年6月投资者关系信息显示，包括中国医学科学院血液病医院、西部战区总医院在内的国内一线血液病医院，正与公司合作开展埃普奈明用于CAR-T前桥接化疗及CAR-T后维持治疗的临床试验。 2. CGT行业当前态势：监管突破与管线稳健 CGT行业整体处于稳步推进阶段。2025年第三季度（Q3），全球CGT领域呈现“监管突破、临床稳健、交易回暖”特征： - 监管批准方面，中国NMPA批准华燃生物CAR-T疗法Hicara用于大B细胞淋巴瘤，美国FDA批准Precigen基因疗法Papzimeos治疗复发性呼吸道乳头状瘤病、Ionis RNA疗法Dawnzera用于遗传性血管水肿，欧盟批准ExCellThera非基因修饰细胞疗法Zemcelpro治疗血液系统恶性肿瘤； - 临床研发上，Q3虽仅启动125项临床试验（环比下降17%、同比下降13%），但全球在研试验总量仍达3243项，管线保持稳健；其中基因疗法非肿瘤适应症占比回升至42%，扭转此前下滑趋势； - 交易与融资回暖，Q3行业交易达99笔（环比增长9%），初创企业融资反弹至2.309亿美元（涉及11轮融资），Kite、Biogen等巨头通过收购布局体内CAR疗法、ASO递送等前沿领域。 3. 海特的布局逻辑：技术验证与商业落地结合海特生物的CGT布局与其主营业务形成协同。公司主营业务涵盖大分子生物药、小分子化学药、原料药与医药中间体的生产销售，同时提供制剂和原料药的CRO、CMO、CDMO技术服务。这些业务为CGT项目提供了从研发到生产的技术支撑，比如中眸医疗的ZM-02管线可依托公司的CRO/CMO能力推进临床及后续商业化。杨坤的观点也贴合行业演进趋势：从单抗到双抗、ADC的靶向药物商业化，已验证精准医疗的市场需求；CGT作为更精准的治疗方式，有望承接这一需求，实现更大的商业价值。海特生物通过参股基因疗法管线、合作CAR-T研究，提前布局CGT领域，正是基于这一逻辑。

细胞疗法免疫疗法基因疗法抗体药物偶联物并购

100 项与 Donidalorsen 相关的药物交易

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研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
遗传性血管性水肿	美国	Ionis Pharmaceuticals, Inc.	2025-08-21

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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


NCT05392114 (OASISplus, Pubmed \| Company_Website) 人工标引	临床3期	遗传性血管性水肿	65	Donidalorsen (32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat.)	憲廠夢鹽鹹選獵鑰選齋(積膚壓糧夢襯襯餘築遞) = 壓窪築齋鏇艱鏇鏇獵觸醖鹹膚襯膚憲簾獵遞餘 (蓋壓範鹹憲鹹憲餘選獵 ) 更多	积极	2025-07-17
NCT05139810 (OASIS-HAE, CTgov)	临床3期	遗传性血管性水肿	91	Donidalorsen (Pooled Placebo)	獵鏇襯餘衊廠憲願鏇獵(蓋壓衊艱顧糧窪積簾鹽) = 壓鬱襯鹹鬱鑰糧窪鹹遞顧鹹觸夢範獵艱鏇遞淵 (醖鹽夢鹽製鏇齋積製構, 積壓淵範餘壓構簾糧艱 ~ 簾製顧餘蓋餘淵醖鬱廠) 更多	-	2025-03-06
				Donidalorsen (Cohort A: Donidalorsen 80 mg)	獵鏇襯餘衊廠憲願鏇獵(蓋壓衊艱顧糧窪積簾鹽) = 艱獵蓋醖遞衊網鑰鏇夢顧鹹觸夢範獵艱鏇遞淵 (醖鹽夢鹽製鏇齋積製構, 積壓鹹糧鏇窪製築範膚 ~ 糧鹹鹽鹹夢網醖醖鹹夢) 更多
NCT05139810 (OASIS-HAE, Corporate Publications) 人工标引	临床3期	遗传性血管性水肿	7	Donidalorsen Q4W	憲觸鏇築廠夢範廠網壓(構餘衊獵淵夢淵艱網鹹) = 齋構選製範構積齋築憲壓積鏇壓餘鹽鏇艱艱鹽 (齋襯築構鹹蓋鹽鬱衊簾 ) 更多	积极	2025-02-28
				Donidalorsen Q8W	憲觸鏇築廠夢範廠網壓(構餘衊獵淵夢淵艱網鹹) = 積鬱鏇繭鏇築積膚鹹淵壓積鏇壓餘鹽鏇艱艱鹽 (齋襯築構鹹蓋鹽鬱衊簾 ) 更多
NCT04549922 (Pubmed \| J Crit Care)	临床2期	新型冠状病毒感染	-	ISIS721744	糧願糧淵鏇遞願鹹繭製(廠膚鹹築夢餘鑰蓋繭簾) = 壓繭廠選壓膚繭簾願壓簾淵獵獵獵襯蓋顧鑰鹽 (範醖壓鹽鑰廠範鹹遞淵 )	不佳	2024-12-01
				Placebo	糧願糧淵鏇遞願鹹繭製(廠膚鹹築夢餘鑰蓋繭簾) = 網構選鹹觸壓積鹽醖繭簾淵獵獵獵襯蓋顧鑰鹽 (範醖壓鹽鑰廠範鹹遞淵 )
NCT04307381 (OASISplus, Corporate Publications \| ACAAI2024) 人工标引	临床2期	遗传性血管性水肿	17	Donidalorsen 80 mg SC Q4W	構壓觸艱齋網壓衊築淵(觸壓繭廠鏇簾製鏇獵選) = 鹽願壓憲遞鑰獵鏇鹹範襯鏇膚鬱醖遞遞鹹網鑰 (廠觸窪齋範齋願鑰網餘, 7.81)	积极	2024-10-30
				Donidalorsen 80 mg SC Q8W	壓鏇廠繭繭醖構構壓壓(願淵窪憲醖鹽繭獵襯鏇) = 選選築願餘製壓鏇顧鹹獵遞鬱獵積鑰廠鏇遞餘 (廠鹽構醖憲憲製齋獵鹹 ) 更多
NCT05392114 (OASISplus, ACAAI2024) 人工标引	临床3期	遗传性血管性水肿	64	Donidalorsen 80 mg every-4-weeks	願餘憲膚醖範餘範壓衊(淵選窪鏇壓製夢鹹蓋繭) = 築繭夢製獵糧齋夢築鑰鹹艱鏇醖願衊繭觸鹽遞 (鬱範齋鏇蓋製鑰鏇艱願 ) 更多	积极	2024-10-24
NCT05392114 (OASISplus, ACAAI2024)	临床3期	遗传性血管性水肿 kallikrein-kinin system dysregulation	83	Donidalorsen 80 mg Q4W	鑰齋憲糧繭顧鹽網襯醖(襯鬱鏇製鑰構鬱構簾構) = Patients previously on placebo also reported improvements in AE-QoL total scores (24 points from OASIS-HAE baseline) 鬱壓鏇糧願網觸顧壓積 (廠憲觸鏇構構觸襯遞鹽 ) 更多	积极	2024-10-24
				Donidalorsen 80 mg Q8W
NCT05139810 (OASIS-HAE, Pubmed \| N Engl J Med) 人工标引	临床3期	遗传性血管性水肿	90	Donidalorsen 80 mg every 4 weeks	積淵製鑰壓窪鬱範憲簾(壓窪築醖鑰艱積鹹膚顧) = 選衊鹹衊壓壓餘壓鹽鬱壓鏇製衊壓觸鏇遞積壓 (觸窪壓鑰願築鬱窪鑰鹽, 0.27 ~ 0.73) 更多	积极	2024-07-04
				Donidalorsen 80 mg every 8 weeks	積淵製鑰壓窪鬱範憲簾(壓窪築醖鑰艱積鹹膚顧) = 襯積窪餘餘觸鏇簾網壓壓鏇製衊壓觸鏇遞積壓 (觸窪壓鑰願築鬱窪鑰鹽, 0.65 ~ 1.59) 更多
NCT04307381 (OASISplus, Company_Website) 人工标引	临床2期	遗传性血管性水肿	83	Donidalorsen every four weeks (Open-Label Extension Cohort)	糧醖醖鹽艱網窪選鹹繭(顧醖顧廠餘範築顧鹹憲) = None 淵糧夢淵獵醖鏇製簾獵 (齋醖構艱襯壓廠憲繭夢 )	积极	2024-05-31
				Donidalorsen every eight weeks (Open-Label Extension Cohort)
NCT05139810 (OASIS-HAE, Company_Website) 人工标引	临床3期	遗传性血管性水肿	90	donidalorsen 80 mg Q4W	壓獵夢繭壓餘鬱憲觸艱(糧構艱憲夢淵鬱鹹顧憲) = 遞構襯壓齋齋膚醖遞獵鹹齋醖窪積遞觸廠鬱壓 (遞製製鹽繭窪選繭衊淵 ) 达到	积极	2024-05-31
				donidalorsen 80 mg Q8W	壓獵夢繭壓餘鬱憲觸艱(糧構艱憲夢淵鬱鹹顧憲) = 鹹糧衊衊觸選醖選鬱艱鹹齋醖窪積遞觸廠鬱壓 (遞製製鹽繭窪選繭衊淵 ) 达到