Therapeutic delivery最新文献

Preparation of artificial tears with in-situ gelling properties for the management of dry eye disease: efficient droplet administration by a 3D-printed device. 具有原位胶凝特性的用于干眼病管理的人工泪液的制备：通过3d打印设备的高效液滴管理。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-20 DOI: 10.1080/20415990.2026.2742651

Sana Yousaf, Saman Zafar, Muhammad Majid Bashir, Sadia Jafar Rana, Jahanzeb Mudassir, Amjad Hussain, Nasir Abbas, Enas Al-Ani, Muhammad Sohail Arshad, Zeeshan Ahmad

{"title":"Preparation of artificial tears with in-situ gelling properties for the management of dry eye disease: efficient droplet administration by a 3D-printed device.","authors":"Sana Yousaf, Saman Zafar, Muhammad Majid Bashir, Sadia Jafar Rana, Jahanzeb Mudassir, Amjad Hussain, Nasir Abbas, Enas Al-Ani, Muhammad Sohail Arshad, Zeeshan Ahmad","doi":"10.1080/20415990.2026.2742651","DOIUrl":"https://doi.org/10.1080/20415990.2026.2742651","url":null,"abstract":"Aims: The present study was aimed at developing in-situ gelling artificial tear formulations and a three-dimensional (3D) printed administration aid.Materials and methods: In-situ gelling formulations were prepared using gellan gum and hydroxypropyl methylcellulose (HPMC). Administration aid was developed by fused deposition modeling approach. Formulations were evaluated for physicochemical properties, sterility, in-vitro erythrocyte morphology assessment, disintegration, in-silico analysis, ex-vivo ocular surface retention, in-vivo ocular irritation and efficacy in dry eye disease model in rabbits. 3D printed device was evaluated for volume of dispensed droplets, usability, instillation success rate and acceptance by human participants.Results: Prepared formulations exhibited acceptable physicochemical properties, sterility and did not irritate the rabbit eye. In-silico analysis suggested interactions between gellan gum, HPMC and ocular proteins (mucin, p-glycoprotein, OATP2A1). Formulations showed an ocular retention time of ~20-24 hours and disintegrated within the same timeframe. A two-fold increase was recorded in the Schirmer 1 test score of formulations treated rabbit eye than the untreated counterpart. A ~ 21% increase in the droplet volume was observed in the presence of administration aid. 3D printed aid facilitated instillation of formulations.Conclusions: Schirmer 1 test scores increased following treatment with prepared formulations as compared to untreated counterparts. An administration aid was successfully engineered for dispensing prepared formulations.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-13"},"PeriodicalIF":3.4,"publicationDate":"2026-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149228478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

An integrated multi-effect microsphere by body temperature assists for transcatheter arterial embolization in hepatocellular carcinoma. 体温综合多效微球辅助肝细胞癌经导管动脉栓塞。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-19 DOI: 10.1080/20415990.2026.2742637

Zhao-Ping Yan, Yongping Yan, Xiangjun Yan, Feng Han, Wen-Gong Yu

{"title":"An integrated multi-effect microsphere by body temperature assists for transcatheter arterial embolization in hepatocellular carcinoma.","authors":"Zhao-Ping Yan, Yongping Yan, Xiangjun Yan, Feng Han, Wen-Gong Yu","doi":"10.1080/20415990.2026.2742637","DOIUrl":"https://doi.org/10.1080/20415990.2026.2742637","url":null,"abstract":"Aims: To develop novel iodine alginate calcium microspheres (IACMs) with intrinsic radiopacity, embolic capability, and body-temperature-assisted iodine conversion for anti-tumor effects.Materials and methods: IACMs were synthesized via Ca2+ crosslinking based on the I- + I2 = I3- equilibrium. Physicochemical characterization was performed. Radiopacity was assessed in vitro and in vivo using digital subtraction angiography and computed tomography (CT). Embolization efficacy was evaluated in rabbit ear artery, normal liver, and VX2 HCC models (n = 5 per group). Cell viability was assessed using a CCK-8 assay.Results: IACMs achieved an encapsulation efficiency of 79.31% ± 2.08%, iodine loading rate of 44.78%. The median particle size (D50) was 176.6 µm. IACMs exhibited good X-ray visibility (in vivo/vitro) with CT attenuation of 4236 ± 48 HU. In the rabbit VX2 HCC model, IACMs produced sustained tumor hyperdensity with partial resolution of the lesion by day 7 (p < 0.001 vs control). In vitro, IACM extract reduced HepaRG and HepG2 cell viability to 8% and 10% at 72 h, respectively (p < 0.001), while direct contact showed no cytotoxicity (>98% viability).Conclusions: IACMs integrate radiopacity, embolization, and temperature-assisted iodine conversion into a single platform, enabling real-time procedural guidance and noninvasive follow-up without exogenous contrast. IACMs show substantial potential for clinical translation in tumor embolization therapy.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-15"},"PeriodicalIF":3.4,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149220648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Bacteria-nanomaterial co-delivery systems for precision therapy: interfacial design, transport behavior, and translational barriers. 用于精确治疗的细菌-纳米材料共递送系统：界面设计，运输行为和翻译障碍。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-19 DOI: 10.1080/20415990.2026.2750073

Xianyu Fan, Ling Mei

引用次数: 0

Alkalizer-based gastroretentive floating tablets of amoxicillin for localized gastric delivery. 碱基胃保留阿莫西林漂浮片用于胃局部输送。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-18 DOI: 10.1080/20415990.2026.2742653

Rahul K, Sarath Chandran C, Reshma K, Gopika Nath C, Abhijith K Shaji, Hiba K, Minil Mukundan, Sreejith K R, Alan Raj, Rajesh Sreedharan Nair, Angel Treasa Alex

{"title":"Alkalizer-based gastroretentive floating tablets of amoxicillin for localized gastric delivery.","authors":"Rahul K, Sarath Chandran C, Reshma K, Gopika Nath C, Abhijith K Shaji, Hiba K, Minil Mukundan, Sreejith K R, Alan Raj, Rajesh Sreedharan Nair, Angel Treasa Alex","doi":"10.1080/20415990.2026.2742653","DOIUrl":"https://doi.org/10.1080/20415990.2026.2742653","url":null,"abstract":"Introduction: Helicobacter pylori infection is a leading cause of peptic ulcer disease, yet amoxicillin trihydrate (AMX) efficacy is limited by its acid-catalyzed degradation and rapid gastric emptying. This study aimed to develop alkalizer-based gastroretentive floating tablets to enhance localized gastric-delivery and improve AMX stability under gastric conditions.Materials and methods: Floating tablets were prepared by direct compression using HPMC K100M, sodium-bicarbonate and microcrystalline-cellulose. A 23 factorial-design (RStudio-v4.3.3) was used to optimize microenvironmental pH, floating lag time (FLT) and drug release. Optimized tablets were characterized for micromeritic properties, XRD, SEM, in vitro buoyancy, swelling, drug release, in vitro anti-H. pylori activity and in vivo gastric retention in Sprague-Dawley rats.Results: The optimized formulation achieved a microenvironmental pH of 7.20 ± 0.26, FLT of 15.84 ± 0.50 s, and total floating time of approximately 12 h. Cumulative AMX release reached 98.49 ± 1.69% over 12 h with non-Fickian kinetics, and XRD/SEM analysis indicated reduced crystallinity and increased amorphous character of AMX within the hydrated matrix. The formulation showed significant in vitro anti-H. pylori activity and prolonged in vivo gastric retention.Conclusion: Alkalizer-based gastroretentive floating AMX tablets combined microenvironmental pH modulation with sustained gastric-retention, supporting their potential as a localized strategy for improved H. pylori eradication.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-17"},"PeriodicalIF":3.4,"publicationDate":"2026-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149216781","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Process-design-translation interplay in lipid nanocarriers for vaginal mucosa delivery: how far have we advanced? 阴道粘膜输送脂质纳米载体的工艺-设计-翻译相互作用：我们进展到什么程度？

IF 3.4

Therapeutic delivery Pub Date : 2026-10-15 DOI: 10.1080/20415990.2026.2747590

Manoj Dalabehera

{"title":"Process-design-translation interplay in lipid nanocarriers for vaginal mucosa delivery: how far have we advanced?","authors":"Manoj Dalabehera","doi":"10.1080/20415990.2026.2747590","DOIUrl":"https://doi.org/10.1080/20415990.2026.2747590","url":null,"abstract":"Nowadays, translational research all across the globe has increasingly been valued for focusing on mucosa delivery across the vaginal avenue, attributed to its anatomy and physiology; however, rapid mucus turnover, poor permeability, and retention popped out as the biggest constraints in this topic. Despite sidestepping these pitiable challenges, lipid nanocarriers (LNCs) are still underexplored and demand upcoming clinical demonstration to bridge the gaps between lab-human vaginal trials. Therefore, this paper delves into a process-design-translation perspective, linking vaginal barriers and lipid/excipient ratio with fabrication strategies and nanocarrier architecture. The first portion critically analyzes the key design/fabrication factors that interplay with LNCs' architecture. In the second portion, various characterization evaluation models of LNCs, viz., quality by design (QbD), lipid/excipient ratio, drug release, vaginal mucoadhesion/penetration (>10-fold higher retention), and cytotoxicity profile, have been discussed with industrial considerations and in vitro-in vivo correlation (IVIVC). Looking ahead into 5-15 years, the author suggests that hybrid LNCs, which may outperform conventional LNCs, and the integration of in-silico models could desirably enhance efficacy. Although considerable, the preclinical model doesn't outline strong enough evidence regarding optimized LNCs-vaginal mucosa delivery. A critical literature analysis was conducted through ScienceDirect, Taylor & Francis, Nature, and PubMed engines till March 2026.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-22"},"PeriodicalIF":3.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184273","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Mechanistic insights into nanostructured lipid carriers: from lipid architecture to drug release behavior. 纳米结构脂质载体的机械洞察：从脂质结构到药物释放行为。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-15 DOI: 10.1080/20415990.2026.2748226

Ritu Kadyan, Renu Kadian, Parijat Pandey, Suman Rohilla

{"title":"Mechanistic insights into nanostructured lipid carriers: from lipid architecture to drug release behavior.","authors":"Ritu Kadyan, Renu Kadian, Parijat Pandey, Suman Rohilla","doi":"10.1080/20415990.2026.2748226","DOIUrl":"https://doi.org/10.1080/20415990.2026.2748226","url":null,"abstract":"Nanostructured lipid carriers (NLCs) are advanced lipid-based nanocarriers. They were developed to overcome the limitations associated with solid lipid nanoparticles (SLNs). Traditional SLNs often face problems such as the risk of drug expulsion during storage and low drug loading due to lipid crystallization. NLCs incorporate a combination of solid and liquid lipids in their matrix, with surfactants used to stabilize the particles. This combination creates a less ordered structure with small imperfections in the matrix, which can improve drug loading and reduce drug expulsion. Relevant literature was searched in PubMed, Google Scholar, Scopus, and Web of Science for publications from 2021 to 2026. This review focuses on how the design of the lipid matrix influences the performance of NLCs. The paper also discusses important physicochemical factors such as partitioning behavior and drug solubility in lipids, which play a key role in drug-lipid interactions. In addition, the relationship between formulation variables, lipid matrix organization, and overall performance is explained. A better understanding of these relationships, along with the mechanisms involved in drug absorption, can support the rational design of NLC formulations. The aim is to help researchers develop lipid-based nanocarriers that are more stable and effective for different drug delivery applications.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-20"},"PeriodicalIF":3.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184232","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Insights into the recent advancements of cephalosporins and their nanoformulations in antibacterial applications. 深入了解头孢菌素及其纳米配方在抗菌应用中的最新进展。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-15 DOI: 10.1080/20415990.2026.2748233

Neelam Sharma, Anshika Tonk, Sukhbir Singh, Ladli Kishore, Ritu Gulia, Sumeet Gupta

{"title":"Insights into the recent advancements of cephalosporins and their nanoformulations in antibacterial applications.","authors":"Neelam Sharma, Anshika Tonk, Sukhbir Singh, Ladli Kishore, Ritu Gulia, Sumeet Gupta","doi":"10.1080/20415990.2026.2748233","DOIUrl":"https://doi.org/10.1080/20415990.2026.2748233","url":null,"abstract":"Cephalosporins are extensively utilized β-lactam antibiotics for the treatment of respiratory, skin, soft tissue, urinary tract, and systemic bacterial infections. However, their extensive and inappropriate use has augmented antimicrobial resistance, reducing their clinical effectiveness. This review recapitulates the physicochemical properties, antibacterial mechanisms, and the challenges of the development of resistance to cephalosporins. Bacteria can develop resistance via pathways, including production of β-lactamase enzymes, mutations in penicillin-binding proteins, efflux pumps, and reduced aqueous-filled protein channels in the outer membrane, which restrict cephalosporin entry. The literature survey was conducted in PubMed, Google Scholar, and ScienceDirect using relevant terms. The current review elucidates the role of nanoparticles in overcoming antibiotic resistance to cephalosporins. Lipid-based, polymeric, inorganic, and hybrid nanocarriers can help avoid antibiotic resistance by disrupting bacterial membrane defenses; limiting drug resistance mechanisms, such as blocking efflux pumps and inactivating β-lactamases; reducing biofilm formation; providing targeted drug delivery; and enhancing solubility. The nanoformulations of first- to fourth-generation cephalosporins demonstrate improved pharmacokinetic performance and therapeutic outcomes against multidrug-resistant pathogens, whereas studies on fifth-generation cephalosporins remain limited. The primary challenges that hinder clinical translation of cephalosporin-based nanoformulations include β-lactam instability, formulation scalability, regulatory approval, low drug loading, and long-term safety.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-15"},"PeriodicalIF":3.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149184308","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Nanostructured formulations of abiraterone and abiraterone acetate: therapeutic potential, pharmacokinetic considerations and bioanalytical perspectives. 阿比特龙和醋酸阿比特龙的纳米结构制剂：治疗潜力、药代动力学考虑和生物分析观点。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-12 DOI: 10.1080/20415990.2026.2742645

Giovanna Capaldi Fortunato, Jonatas Lobato Duarte, Marcela Tavares Guiguer, Marlus Chorilli

{"title":"Nanostructured formulations of abiraterone and abiraterone acetate: therapeutic potential, pharmacokinetic considerations and bioanalytical perspectives.","authors":"Giovanna Capaldi Fortunato, Jonatas Lobato Duarte, Marcela Tavares Guiguer, Marlus Chorilli","doi":"10.1080/20415990.2026.2742645","DOIUrl":"https://doi.org/10.1080/20415990.2026.2742645","url":null,"abstract":"Abiraterone acetate (AbA) is widely used in the treatment of metastatic castration-resistant prostate cancer. After administration, the prodrug is rapidly converted into its active metabolite, abiraterone (Ab). However, oral administration is compromised by an unfavorable biopharmaceutical profile because the drug has very low solubility in water and absorption which is restricted by dissolution, plus a marked food effect that causes substantial variability in pharmacokinetics. This review examines the therapeutic potential of nanostructured drug delivery systems designed to overcome these drawbacks. Concerning sustained intracellular exposure and co-delivery strategies, polymeric nanoparticles such as those made from PLGA are considered. Lipid-based nanocarriers, including solid lipid nanoparticles and nanostructured lipid carriers, are evaluated with respect to their matrix structure, physicochemical stability, and digestion-dependent behavior that affects intestinal absorption. Nanoamorphous AbA systems are also looked at as a way of improving dissolution, it being necessary to use strict solid-state stabilization in order to prevent recrystallization. Moreover, the review looks at the bioanalytical issues that are relevant to the interpretation of the performance of various formulations, with a focus on LC-MS/MS methods for quantifying AbA, Ab and the related metabolites in biological matrices. The methodological priorities required for clinical translation are identified, among them standardized biorelevant testing and pharmacokinetic study designs that are able to take into account inter-individual variability and food effects. Literature searches were carried out in PubMed and the Web of Science Core Collection, with additional input from ScienceDirect and by screening the reference lists, for publications up to July 2026.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-23"},"PeriodicalIF":3.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149166789","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Methodical design, development, and evaluation of transferosomal transdermal films for enhanced delivery of lornoxicam and thiocolchicoside. 有系统地设计、开发和评估转移体透皮膜，以增强氯诺昔康和硫代秋芥子苷的递送。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-10 DOI: 10.1080/20415990.2026.2742634

Gopinath Subramaniyan, Shaik Rubina, Bachu Venkata Ramana, Meriton Stanley A, Devasena Srinivasan

{"title":"Methodical design, development, and evaluation of transferosomal transdermal films for enhanced delivery of lornoxicam and thiocolchicoside.","authors":"Gopinath Subramaniyan, Shaik Rubina, Bachu Venkata Ramana, Meriton Stanley A, Devasena Srinivasan","doi":"10.1080/20415990.2026.2742634","DOIUrl":"https://doi.org/10.1080/20415990.2026.2742634","url":null,"abstract":"Aim: The present work aimed to enhance systemic availability through optimized transferosomal transdermal films of lornoxicam (LXM) and thiocolchicoside (TCS). Transferosomes were prepared using the thin-film hydration method, in which LXM and TCS, which have low oral bioavailability and adverse effects, were loaded.Methods: A Box-Behnken design was used to develop 17 vesicles in which dipalmitoyl phosphatidylcholine (X1), Span-80 (X2), and sonication time (X3) were varied and assessed for size, polydispersity index , and entrapment efficiency. The best vesicles were made into films. Films were extensively tested for physical, in vivo, and in vitro permeation, and skin irritancy characteristics.Results: The findings revealed vesicle sizes of 124 ± 2.25 to 400 ± 1.55 nm and a zeta potential of -48 to -20 mV. The drug entrapment was more than 80% for both drugs. In vitro and ex vivo permeation showed that the deformable vesicle film permeated much better than plain LXM and TCS films, and the permeation was directly proportional to the Aloe vera leaf mucilage (AVLM) and lime oil (LO) concentration. Also, the deformable vesicle films were reported to be devoid of skin irritation.Conclusion: The research finds that deformable vesicle films of LXM/ TCS are promising for effective transdermal delivery.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-15"},"PeriodicalIF":3.4,"publicationDate":"2026-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149166758","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

An evaluation of mitapivat for the treatment of non-transfusion-dependent alpha- and beta-thalassemia. 米他伐特治疗非输血依赖型地中海贫血的评价。

IF 3.4

Therapeutic delivery Pub Date : 2026-10-06 DOI: 10.1080/20415990.2026.2742638

Aaron N Cheng, Hanny Al-Samkari

{"title":"An evaluation of mitapivat for the treatment of non-transfusion-dependent alpha- and beta-thalassemia.","authors":"Aaron N Cheng, Hanny Al-Samkari","doi":"10.1080/20415990.2026.2742638","DOIUrl":"https://doi.org/10.1080/20415990.2026.2742638","url":null,"abstract":"Non-transfusion-dependent thalassemia (NTDT) describes a group of inherited hemoglobin disorders characterized by hemolytic anemia and ineffective erythropoiesis. Although patients with NTDT do not require lifelong regular transfusions for survival, they are at a substantial risk of complications including iron overload, pulmonary hypertension, extramedullary hematopoiesis, and others. Until recently, treatment options were limited to supportive care, intermittent transfusions, occasional splenectomy, and off-label therapies. Mitapivat, an oral activator of erythrocyte pyruvate kinase, is the first disease-directed therapy for adults with NTDT to be approved by the U.S. Food and Drug Administration. Mitapivat improves red cell survival and reduces hemolysis by improving erythrocyte energy production through the glycolytic pathway. Its approval was supported by the Phase III ENERGIZE trial, which showed significant improvements in hemoglobin concentration, hemolysis markers, and fatigue among adults with α- and β-thalassemia. In this review, we summarize the rationale for pyruvate kinase activation in thalassemia, present preclinical and clinical evidence supporting the use of mitapivat in NTDT, discuss the efficacy and safety profile of mitapivat, and review practical considerations for prescribing. We also discuss knowledge gaps and the need for long-term follow-up studies to clarify the role of mitapivat within the changing therapeutic landscape for thalassemia.","PeriodicalId":22959,"journal":{"name":"Therapeutic delivery","volume":" ","pages":"1-12"},"PeriodicalIF":3.4,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149115050","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0