There is considerable morbidity and mortality associated with cardiac surgery. Currently little effort is made to quantify how well the immune system of an individual can cope with inflammation or infection to which they are exposed during surgery.
The investigators have previously demonstrated that having higher pre-operative antibody levels is associated with a lower risk of infection and a shorter stay in hospital after cardiac surgery.
The investigators aim to study 150 patients undergoing aortic valve replacement and explore their dynamic immune responsiveness. The investigators will determine if this response is correlated with the post-operative outcome (development of post-operative infection or increased length of hospital stay).
The investigators will compare this response with the previously measured static markers of immune competence and also with a novel device that may give a more rapid measure of dynamic immunity.
The investigators will approach patients in the cardiac surgical pre-assessment clinic to see if they are willing to participate in the study.
Immediately once under anaesthetic blood will be taken for testing and then again at the end of surgery, 24h after surgery, at discharge from hospital, and at follow-up clinic approximately 4 weeks later. There will be no additional needle insertions on top of those routinely performed. The investigators will collect data from the routine observations as far as 1 year after surgery.
If the investigators can show an association between immune function and subsequent post-operative outcome it may be possible to determine ways to improve outcomes for patients undergoing heart surgery.
This might include better information on risks and benefits of surgery, actively boosting immune function (vaccination, immune-nutrition), passively improving immunity (administering antibodies), or consider current alternatives to open heart surgery where the threat of infection or inflammation may be markedly reduced (eg trans-catheter aortic valve implantation)

开始日期2025-09-01

申办/合作机构

Barts & The London NHS Trust

[+1]

NCT06778122

/ Not yet recruitingN/AIIT

A Series of N-of-1 Randomised, Adaptive, Blinded, Placebo-Controlled Trials of Overnight In-Bedroom Air Filtration As Adjuvant Treatment in Reducing Inflammatory Biomarkers Among Survivors of Adult-Onset Cancer with Elevated C-Reactive Protein

The BREATHS trial aims to investigate whether overnight in-bedroom air filtration reduces inflammation and cardiac biomarkers in adult survivors of cancer who are at high risk for cardiovascular complications.
The study consists of individualized experiments (N-of-1 trials) conducted in the home settings of adults residing in densely populated urban areas with the most severe air quality levels in Valencia, Spain, where fine particulate matter (PM2.5) and nitrogen dioxide levels exceed the limits set by the World Health Organization (WHO) and EU Directive.
Participants will be exposed to 3 treatment sets (blinded phase), each consisting of a 14-day period of filtered air using a portable air filtration unit ("true-HyperHepa) and a 14-day period of unfiltered air (using the same portable unit with "sham filters"). The intervention will be administered nightly for a minimum of 7 consecutive hours and will last between 4 and 12 weeks for each participant, contingent upon the demonstration of clinical benefit, defined by a reduction in the levels of the blood biomarker C-reactive protein. An unblinded phase will be implemented for participants who do not experience a clinically meaningful change in CRP. During this phase, they will undergo a 14-day period without treatment, followed by 14-day period of both nightly and daily filtered air therapy.
The primary endpoint of this study is defined as the change in blood concentrations of CRP. The secondary endpoints include the changes in levels of three other noninvasive blood biomarkers associated with inflammation and cardiovascular health, and blood pressure.
Both indoor and outdoor fine particulate matter (PM2.5) exposure concentrations will be continuously monitored in the study.

开始日期2025-09-01

申办/合作机构

University College London

ISRCTN16721218

/ RecruitingN/AIIT

The emotional impact of restoring sight (EIRS): well-being and mental health in children and adolescents receiving sight restoring therapy for eye disease

开始日期2025-09-01

申办/合作机构

University College London

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0 项与 University College London 相关的专利（医药）

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156,399

项与 University College London 相关的文献（医药）

2026-01-01·Neural Regeneration Research

Shared mechanisms and pathological phenotypes underlying aminoacyl-tRNA synthetase-related neuropathies

Article

作者: Rhymes, Elena R. ; Sleigh, James N.

2025-12-31·Human Fertility

Oocyte donation pregnancy: What do women understand about risk? an interview study of women who underwent oocyte donation pregnancy

Article

作者: Bains, Saibhan ; Nicholls, Jacqueline ; Whitten, Melissa ; Maslowski, Katherine ; Lanceley, Anne

Patient consent is a fundamental legal and professional requirement of any intervention offered to a patient and must include discussion of risks and benefits. The number of women becoming pregnant via oocyte donation has increased markedly in recent years. Such pregnancies are accompanied by different risks to those of naturally conceived pregnancies. Anecdotal evidence suggests that women seeking oocyte donation pregnancy gain limited appreciation of the risks attending oocyte donation during consent discussions. To explore women's understanding of the risks associated with oocyte donation pregnancy a qualitative interview study was conducted in an ante-natal facility in an urban hospital. Sixteen women who had experienced oocyte donation pregnancy participated in semi-structured, audio-recorded interviews exploring their experience of the consent process for oocyte donation and their awareness of pregnancy-related risks once pregnant. Data were analysed using thematic analysis. Our principal finding was that women were unaware that their oocyte donation pregnancy carried higher risks than a typical IVF pregnancy. Three themes emerged: (1) women tended to care most about risks associated with success/failure of oocyte donation; (2) women did not know that oocyte donation pregnancies carry specific risks above and beyond IVF pregnancies; (3) women were ambivalent towards receiving risk information. For women accepting an oocyte donation consent at the time of undergoing IVF should include discussion of the risks of subsequent pregnancy to ensure that women are fully informed. Women's understanding of risks should be routinely checked ante-natally.

2025-12-31·Epigenetics

Differential methylation in blood pressure control genes is associated to essential hypertension in African Brazilian populations

Article

作者: Magalhães Borges, Vinícius ; Avila Martins, Camila Cristina ; Kimura, Lilian ; Mingroni-Netto, Regina Célia ; Alvizi, Lucas ; Maschietto, Mariana ; Victorino Krepischi, Ana Cristina ; Nunes, Kelly

While genetic studies have provided insights into essential hypertension (EH, defined by high blood pressure ≥140/90 mmHg), investigation through epigenetics may address gaps in understanding its heritability. This study focused on African Brazilian populations in Vale do Ribeira River region, due to their high hypertension prevalence. We aimed to determine if DNA methylation is linked to hypertension susceptibility, through a genome-wide evaluation of 80 peripheral blood samples from normotensive (39) and hypertensive (41) individuals, with Infinium Methylation EPIC BeadChip platform. Data were analyzed using ChAMP package and cross-referenced with information from databases such as EWAS Atlas, GWAS catalog, GeneCards, literature, and tools such as VarElect and EWAS Toolkit. The comparison between hypertensive and normotensive revealed 190 differentially methylated CpG positions (DMPs) and 46 differentially methylated regions (DMRs), both with p-value ≤0.05. Among the DMPs, 27 were found to have a plausible role in blood pressure. Among the DMRs, those mapped to ABAT, BLCAP, CERS3, EIF4E, FMN1, GABBR1, HLA-DQB2, HOXA5, IL5RA, KCNH2, MIR487B, MIR539, MIR886, MKRN3, NUDT12, PON3, RNF39, RWDD3, and TSHBS1 were highlighted because of their lowest p-values, current literature, and/or VarElect prioritization. Our findings suggest that differences in methylation contribute to the high susceptibility to essential hypertension in these populations.

889

项与 University College London 相关的新闻（医药）

2025-05-06

·学术经纬

《细胞》：阿尔茨海默病让人健忘，罪魁祸首找到了！

说到阿尔茨海默病，很多人知道它会让人变得越来越健忘，最终丧失记忆，变得“痴呆”。而大脑认知功能逐步衰退的背后，是神经细胞的功能障碍和逐渐死亡，首当其冲的就是与学习、记忆能力密切相关的脑区，比如海马区。经过几十年的研究，科学家在阿尔茨海默病患者的大脑中发现，有两种毒性蛋白对神经元的影响最为明显：β-淀粉样蛋白（Aβ）聚集而成的斑块和病理性tau蛋白造成的神经原纤维缠结。图片来源：123RF基于这种标志性的病理特征，一些已经获批或正在开发的阿尔茨海默病疗法通过消除患者脑中的Aβ斑块来延缓疾病发展。另一方面，也有一些研究致力于减轻tau病理来开发有效的阿尔茨海默病疗法，但由于tau蛋白本身作为微管相关蛋白具有正常的生理功能，直接靶向全部tau蛋白的策略可能带来严重副作用，有必要寻找具有致病性作用的tau形式并开发相应的治疗策略。最近，英国伦敦大学学院的研究人员在《细胞》上发表论文，揭示了一种特定类型的tau蛋白，对于海马神经元之间的正常交流具有破坏作用。这意味着，这类特殊的tau蛋白——他们称为可溶性高分子量tau（HMW tau），可能是在阿尔茨海默病早期造成患者健忘、认知能力下降的一个关键原因。海马神经元之间的正常交流依靠电信号的有序发放，在电生理记录下表现为单个的动作电位或一连串的动作电位。在这项研究中，科学家们使用前沿的高分辨率体内探针，在小鼠清醒、运动等不同状态时记录到海马神经元发放动作电位的模式和各类参数，从而解码海马神经元之间的交流。这些小鼠有一部分经过基因操作模拟了人类阿尔茨海默病患者的基因特征，并会随着年龄增长在脑中出现Aβ病理或是tau蛋白缠结或是这两种病理特征。▲野生型小鼠和阿尔茨海默病模型小鼠的海马神经元发放动作电位的模式不同（图片来源：参考资料[1]）比较不同小鼠的海马神经元放电特征后，研究人员发现，即使没有Aβ的影响，仅仅是tau蛋白就会让神经元爆发动作电位的特征发生异常，尤其是会显著抑制神经元自发的“爆发式放电模式”。而海马神经元这种爆发式的连续多次快速放电模式，对于记忆的形成十分重要。这也就意味着，tau蛋白可以通过干扰海马神经元的高频放电模式直接影响大脑的记忆回路。▲没有Aβ的影响时，仅tau蛋白也会特异性破坏海马神经元的爆发式放电活动（图片来源：参考资料[1]）分析还发现，这些受tau影响的海马神经元同时还有钙通道CaV2.3表达降低的现象，可以解释为什么爆发式放电模式会受损。并且，当研究者在转基因小鼠中用药物抑制可溶性tau蛋白的产生时，海马神经元中CaV2.3的表达显著增加，表明这种影响神经元高频放电模式的钙通道受损与可溶性tau蛋白有关。研究人员尤其注意到一种可溶性、高磷酸化tau蛋白形式。在处于阿尔茨海默病各个发展阶段的患者中都能观察到这种形式的tau蛋白，尽管它们在总tau蛋白中所占的比例不到1%，但它们能在神经细胞中传播并触发tau蛋白沉积，与患者的临床进展速度密切相关。▲HMW tau蛋白的存在破坏海马神经元之间通过爆发式放电模式交流（图片来源：参考资料[1]）而新研究发现，这类HMW tau也存在于模拟阿尔茨海默病的小鼠脑中，并且一旦通过药物处理减少，可以使动物恢复更快、更复杂的海马神经元放电模式。研究人员进一步从一名已故阿尔茨海默病患者捐献的脑组织中分离提取出HMW tau，将其输注到正常小鼠的脑切片中，发现即便浓度很低，也同样会抑制其海马神经元爆发式放电，验证了这类有害的tau蛋白是破坏神经元爆发式放电能力的罪魁祸首。负责这项研究的Marc Aurel Busche博士认为，这些新发现为治疗阿尔茨海默病开辟了一个有希望的靶点，有望开发新的疗法来保护患者的认知功能。参考资料：[1] Samuel Harris et al., Alzheimer’s disease patient-derived high-molecular-weight tau impairs bursting in hippocampal neurons, Cell (2025), https://doi-org.libproxy1.nus.edu.sg/10.1016/j.cell.2025.04.006[2] Memory forming brain cell activity disrupted by tau protein in Alzheimer’s. Retrieved Apr. 30, 2025 from https://www.ukdri.ac.uk/news-and-events/memory-forming-brain-cell-activity-disrupted-tau-protein-alzheimers欢迎转发到朋友圈，谢绝转载到其他平台。如有开设白名单需求，请在“学术经纬”公众号主页回复“转载”获取转载须知。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。更多推荐点个“在看”再走吧~

细胞疗法

2025-05-01

·Pharmaceutical Technology

New £1bn cancer research hub planned for South London

The new development will sit on a 12-acre plot between the Institute of Cancer Research (ICR) and one of The Royal Marsden’s sites in Sutton. Image credit: Aviva Capital Partners and Socius. Plans have been unveiled for The London Cancer Hub, a state-of-the-art cancer research and laboratory hub to be located in South London, UK. Insurance company Aviva’s investment arm and property developer Socius have submitted a planning application for the space, which will cost around £1bn, to the London Borough of Sutton. The new development will sit on a 12-acre plot in Sutton, nestled between the Institute of Cancer Research (ICR) and one of The Royal Marsden’s sites. The ICR has played a pivotal role in advancing cancer research, being the research institute responsible for linking cigarette use to lung cancer. The Royal Marsden is one of the leading hospitals in Europe for cancer diagnosis, treatment, and care. The London Cancer Hub is expected to complement the cancer expertise already in Sutton. AVIVA and Socius hope to entice leading life sciences companies and promote collaboration with academic researchers and clinicians to drive innovation in cancer research. The new buildings will range from large-scale facilities for bigger pharmaceutical and life science companies, to smaller, flexible lab and incubator spaces to cater for start-ups. There will also be space for wet labs, Good Manufacturing Practice (GMP) facilities, specialist equipment, and office space. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence In addition, a total of 3,000 new jobs will be created by the new hub, mainly in high-skilled R&D and small-scale manufacturing related to life sciences. The ICR welcomed the news, with the institute’s chief executive Professor Kristian Helin commenting: “We’re very pleased to be working with Aviva Capital Partners and Socius on plans to further build the thriving multidisciplinary research district in Sutton, and we’re hoping that the project will attract a range of companies to locate here alongside us, as well as provide a home for companies that spin out from our science in the future. “The London Cancer Hub has great potential to enhance our scientific partnerships with companies in the pharmaceutical and broader life-sciences industries, in areas from the discovery and development of new cancer drugs to the creation of new treatment and diagnostic technologies.” Mayor and government initiative The London Cancer Hub was included as a key district for growth as part of London Mayor Sadiq Khan’s growth plan for the capital. The plan is a blueprint for spurring growth in London, to make its economy £107bn larger in 2035. The London Cancer Hub will also contribute to the plan’s goal of creating over 150,000 jobs by 2028. The new hub is one of many “innovation clusters” being developed across the city, such as Canary Wharf Life Sciences and Healthcare, The Knowledge Quarter, and Olympic Park Innovation District, among others. Pharmaceutical Technology has visited both the Canary Wharf cluster and University College London’s (UCL) site at the Olympic Park, where incumbents showed a clear focus on government plans to bolster the UK life sciences sector. Last month, the UK Government announced almost £14bn in funding for research & development (R&D) in life sciences. Life sciences is one of eight growth sectors being targeted by Prime Minister Keir Starmer as part of his “Plan For Change”.

2025-04-29

·蒲公英Ouryao

一周 | 国际法规药事(04.21-04.27)

◆ ◆ ◆ ◆Paul's Insight2025年4月21日-4月27日◆ ◆ ◆ ◆过去一周（4月21日至4月27日），全球药事监管和行业机构齐齐发声，犹如一场精彩的药品政策与科技盛宴。从FDA的合成色素退市行动，到EMA的罕见病新疗法推荐；从EDQM对生物制品检测标准的升级，到MHRA多款创新疗法的批准；再到行业协会PDA和ISPE在指南与实践层面的多重发力，这一连串新闻不仅彰显了各大机构的监管力度，更映射出制药行业在创新、合规与可持续性间的不断平衡。美国FDA从食药安全到创新药物的“双轨”发力1. 合成食用色素“退场”行动4月22日，美国FDA宣布，计划逐步淘汰美国食品供应中的合成色素（Petroleum-Based Synthetic Dyes），为儿童健康撑起防护伞。FDA表示，这一举措，是“让美国再次健康”（Make America Healthy Again）计划的重要里程碑。此前，1月15日赤藓红（FD&C Red No. 3）禁用已拉开了帷幕，后续这一更广泛的色素退市，将持续推动产业寻求天然、低风险的替代方案。图：赤藓红禁用后，FDA再推新规，八大合成食用色素将退场https://www.fda.gov/news-events/press-announcements/hhs-fda-phase-out-petroleum-based-synthetic-dyes-nations-food-supply2. 药品安全敲响警钟：外用非那雄胺安全红线4月22日，FDA对市场上未经批准的外用非那雄胺配药制剂发出警示，提醒医疗机构、药房及消费者：目前无任何局部非那雄胺产品拥有FDA批准的标签和安全、有效性验证，配制药剂可能隐藏严重风险，包括与米诺地尔等混合制剂的混用场景。FDA直指部分处方药店和远程医疗平台的药品安全风险行为，呼吁行业加强自律与审查。图：就非那雄胺外用安全问题，FDA发布脱发常用药警示https://www.fda.gov/drugs/human-drug-compounding/fda-alerts-health-care-providers-compounders-and-consumers-potential-risks-associated-compounded3. 中国创新药的海外“登台”——派安普利单抗获批4月24日，FDA官网放出重磅消息：康方生物自主研发的PD1单抗派安普利单抗（Penpulimabkcqx）获批，用于复发或转移性非角化型鼻咽癌（NPC）治疗。该产品采用IgG1亚型Fc段改造设计，依托突破性疗法认定、孤儿药资格和快速通道三重加速，联袂正大天晴药业集团布局后续商业化。此举不仅为全球鼻咽癌患者带来曙光，也标志着中国创新药在国际舞台上稳步迈进。（点击此处查看相关阅读）图：中国制药行业又一里程碑，派安普利单抗获FDA批准进入美国市场https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma美国PDA发布新闻季刊4月24日，美国注射剂协会（PDA）发布新闻季刊，总结其在过去一个季度的工作，要点如下。图：PDA发布新闻季刊https://www.pda.org/pda-letter-portal/home/full-article/community-news-quarterly-april-20251）2024年PDA出版奖揭晓PDA颁发奖项给Novartis&Ogilvy团队，表彰其用于无菌性检测的创新方法，该论文名为《一种利用固相流式细胞术进行细胞制备、培养基和缓冲液的快速无菌方法》。年度PDA Letter文章奖项授予武田制药的《理解日本品质》。2）区域分会动态：德国、奥地利和瑞士分会（DACH）亮点纷呈DACH分会“目视检测与颗粒挑战”网络研讨会吸引400余名专家，聚焦AI时代的风险基础方法与质量设计。PDA表示，此次网络研讨会超出预期，如此高的参与度凸显了业界对目视检测和颗粒物相关挑战的关注，同时也强化了DACH分会促进制药行业知识交流和最佳实践的使命。欧盟EMA欧盟罕见病与适应症扩展双管齐下1. DMD新疗法——组蛋白去乙酰化酶（HDAC）抑制剂获“有条件上市许可”4月25日，欧洲药品管理局（EMA）发布杜氏肌营养不良症（DMD）创新药物Duvyzat（givinostat）“有条件上市许可”建议。该组蛋白去乙酰化酶（HDAC）抑制剂在120例6岁以上患者的18个月研究中显著延缓台阶攀爬时间，虽次要终点未全部显著，但整体数据朝积极方向发展。获得“有条件上市许可”，这是欧盟的监管机制之一，旨在促进早期上市药物，以满足未满足医疗需求的。EMA要求申请人后续进行一项随机、安慰剂对照研究，并进行长期随访将补足盲点。上市许可人为Italfarmaco S.p.A.，是是一家总部位于意大利米兰的制药跨国公司。图：杜氏肌营养不良症（DMD）创新药物获“有条件上市许可”https://www.ema.europa.eu/en/news/new-treatment-against-duchenne-muscular-dystrophy2. 甲状腺眼病首创疗法Tepezza获积极审评意见4月25日，EMA对甲状腺眼病（TED）首款靶向IGF1受体的单抗Teprotumumab（Tepezza）给出积极评审意见。三项RCT临床显示活动期患者24周内眼球突出度平均减少2.0–2.3mm，临床活动评分大幅改善；慢性期亦有约1.5mm降低。基于肌肉痉挛、脱发等可管理风险，EMA将附加风险最小化措施并转交欧委会审批。申请人为Amgen Europe B.V.，是全球领先的生物技术公司安进在欧洲的子公司。图：甲状腺眼病首创疗法Tepezza获积极审评意见https://www.ema.europa.eu/en/news/first-treatment-against-severe-thyroid-eye-disease3. CHMP会议要点：新药、扩展、再审与撤回4月22–25日的EMA人药委员会（CHMP）会议，推荐16款新药（包含Vertex囊性纤维化三联疗法、差异化适应症的多款孤用药及九款生物类似药），推动10项适应症扩展，如武田制药的Adcetris多淋巴瘤适应等；同时，礼来的阿尔茨海默药物Kisunla申请再审，Biohaven和辉瑞的两款产品适应失利撤回。整体来看，EMA通过CMA平衡未满足需求与证据要求，也显示出对成熟产品生命周期管理策略的开放态度。图：2025年4月22日至25日人药委员会( CHMP )会议要点https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-22-25-april-2025欧洲EDQM高通量测序入典，提升生物制品安全4月23日，EDQM欧洲药典委员会（EPC）正式采纳通则“2.6.41病毒外源性因子检测的高通量测序（HTS）”，自2026年4月1日起生效。新章节涵盖非靶向与靶向HTS方法学、基因组/转录组数据分析和验证指南，驱动与ICH Q5A(R2)修订及WHO国际参考组协同，助力生物制品从传统动物实验迈向更高灵敏度的分子检测。图：高通量测序入欧洲药典https://www.edqm.eu/en/-/epc-adopts-cutting-edge-hts-chapter-to-enhance-viral-contaminant-detection-in-biological-products英国MHRA创新疗法齐发1. 4月拜耳新药Acoramidis获批，用于心肌病4月25日，MHRA批准拜耳制药的Acoramidis（Beyonttra），用于治疗因变异型或野生型转甲状腺素蛋白淀粉样变性（ATTR-CM）引起的心肌病（心肌损伤）成年患者。，632例国际多中心研究显示30个月心血管住院率和死亡率显著降低，6分钟步行距离改善。口服每日两次，通过IRP机制加速审评。图：拜耳新药Acoramidis获批，用于心肌病https://www.gov.uk/government/news/acoramidis-approved-to-treat-wild-type-or-variant-transthyretin-amyloidosis-in-adults-with-cardiomyopathy2. 白血病CAR-T疗法Obecabtagene autoleucel获有条件上市批准4月25日，obecabtagene autoleucel (Aucatzyl) 这一CART疗法获条件上市许可，用于治疗复发或难治性B 细胞前体急性淋巴细胞白血病 (ALL) 的成年患者。FELIX研究153例数据显示12个月总生存率81%、完全缓解55%，后续需提供更多数据满足全面上市要求。上市许可人为Autolus Therapeutics，是一家总部位于英国的生物制药公司，该公司于2014 年从伦敦大学学院（UCL）分拆成立。图：白血病CAR T疗法Obecabtagene autoleucel获有条件上市批准https://www.gov.uk/government/news/obecabtagene-autoleucel-conditionally-approved-to-treat-adults-with-relapsed-or-refractory-b-cell-precursor-acute-lymphoblastic-leukaemia3. 百健新药在英获批4月23日，MHRA批准了 omaveloxolone（Skyclarys），这是英国首个针对 16 岁及以上患者的治疗药物，用于治疗一种名为弗里德赖希共济失调的罕见神经退行性运动障碍。弗里德赖希共济失调是最常见的遗传性共济失调（由遗传基因引起）。据估计，每5万人中至少有1人患有此病。弗里德赖希共济失调的症状包括平衡和运动障碍，并会随着时间的推移逐渐恶化。该药物为口服胶囊剂。上市许可人为Biogen Netherlands B.V.，为全球领先的生物技术公司 Biogen Inc.（百健公司）在荷兰的全资子公司。图：百健新药在英获批https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-friedreichs-ataxia-omaveloxolone4. 辉瑞血友病药物获批4月22日，MHRA批准辉瑞药物Marstacimab（Hympavzi），用于预防或减少12岁及以上、体重至少35公斤的血友病A和B患者的出血。该药物是同类药物中第一个通过针对血液凝固过程中的蛋白质来发挥作用的药物。Marstacimab每周一次皮下注射，使用预充式注射器或注射笔。患者或护理人员经过适当培训后即可自行注射药物。图：辉瑞血友病药物获批https://www.gov.uk/government/news/marstacimab-approved-to-treat-patients-aged-12-years-and-above-weighing-at-least-35-kg-with-haemophilia-a-or-b国际ISPE发布数字化验证良好实践指南国际制药工程协会（ISPE）发布了《数字化验证良好实践指南》（Good Practice Guide: Digital Validation），为制药企业提供从传统验证向数字化框架过渡提供了指导。这本90页的指导文件，旨在帮助制药企业采用数字化验证工具（Digital Validation Tools, DVT），提升数据可靠性、运营效率和监管合规性。该指南由全球专家团队开发，其核心成员来自于武田制药、葛兰素史克等全球药企和行业咨询公司。ISPE表示，指南结合真实案例和结构化方法，适用于从初创到优化现有系统的各类企业。（点击此处查看相关阅读）图：ISPE最新发布《数字化验证良好实践指南》https://ispe.org/publications/guidance-documents/good-practice-guide-digital-validation结语回望过去一周，FDA、EMA、EDQM、MHRA、PDA和ISPE等监管与行业机构的多项决策、指南与更新，彰显监管严谨，又鼓励技术革新。从食品安全到精准疗法，从分子检测到数字化治理，昭示出全球制药行业正朝着更健康、更高效、更可持续的未来协奏。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月6日 | 线上 | 新法规和应用场景下的全球包材注册策略和最佳实践5月8日 | 线上 | 双维度解锁无菌药品生产污染防控策略

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药物(靶点)	适应症	全球最高研发状态

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