Journal of Controlled Release, Год журнала: 2025, Номер unknown, С. 113837 - 113837
Опубликована: Май 1, 2025
Язык: Английский
Signal Transduction and Targeted Therapy, Год журнала: 2024, Номер 9(1)
Опубликована: Июль 17, 2024
Traditional therapeutic approaches such as chemotherapy and radiation therapy have burdened cancer patients with onerous physical psychological challenges. Encouragingly, the landscape of tumor treatment has undergone a comprehensive remarkable transformation. Emerging fervently pursued modalities are small molecule targeted agents, antibody-drug conjugates (ADCs), cell-based therapies, gene therapy. These cutting-edge not only afford personalized precise targeting, but also provide enhanced comfort potential to impede disease progression. Nonetheless, it is acknowledged that these strategies still harbour untapped for further advancement. Gaining understanding merits limitations holds promise offering novel perspectives clinical practice foundational research endeavours. In this review, we discussed different modalities, including drugs, peptide antibody cell therapy, It will detailed explanation each method, addressing their status development, challenges, solutions. The aim assist clinicians researchers in gaining deeper diverse options, enabling them carry out effective advance more efficiently.
Язык: Английский
Процитировано
219
Chemical Reviews, Год журнала: 2024, Номер 124(3), С. 929 - 1033
Опубликована: Янв. 29, 2024
RNA-based therapies have catalyzed a revolutionary transformation in the biomedical landscape, offering unprecedented potential disease prevention and treatment. However, despite their remarkable achievements, these encounter substantial challenges including low stability, susceptibility to degradation by nucleases, prominent negative charge, thereby hindering further development. Chemically modified platforms emerged as strategic innovation, focusing on precise alterations either RNA moieties or associated delivery vectors. This comprehensive review delves into platforms, underscoring significance augmenting performance translational prospects of therapeutics. It encompasses an in-depth analysis various chemically that been instrumental propelling therapeutics toward clinical utility. Moreover, scrutinizes rationale behind diverse chemical modification techniques aiming at optimizing therapeutic efficacy molecules, facilitating robust management. Recent empirical studies corroborating enhancement through modifications are highlighted. Conclusively, we offer profound insights transformative impact drugs delineates prospective trajectories for future development integration.
Язык: Английский
Процитировано
53
Advanced Drug Delivery Reviews, Год журнала: 2024, Номер 213, С. 115419 - 115419
Опубликована: Авг. 5, 2024
RNA medicines represent a paradigm shift in treatment and prevention of critical diseases global significance, e.g., infectious diseases. The highly successful messenger (mRNA) vaccines against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) were developed at record speed during the disease 2019 pandemic. A consequence this is exceptionally shortened vaccine development times, which combination with adaptability makes technology attractive for pandemic preparedness. Here, we review state art design delivery based on different modalities, including linear mRNA, self-amplifying RNA, trans-amplifying circular RNA. We provide an overview clinical pipeline diseases, present analytical procedures, are paramount characterizing quality attributes guaranteeing their quality, discuss future perspectives using to combat pathogens beyond SARS-CoV-2.
Язык: Английский
Процитировано
14
Vaccines, Год журнала: 2025, Номер 13(1), С. 56 - 56
Опубликована: Янв. 10, 2025
Background: The emergence of more than 40 new infectious diseases since the 1980s has emerged as a serious global health concern, many which are zoonotic. In response, international organizations, including US Centers for Disease Control and Prevention (CDC), World Health Organization (WHO), European Center (ECDC), have developed strategies to combat these threats. need rapid vaccine development been highlighted by Coronavirus disease 2019 (COVID-19), mRNA technology shown promise platform. While acceleration successful, concerns raised about technical limits, safety, supply, distribution vaccines. Objective: This study analyzes status platform in pandemics explores ways respond future pandemic crises through an overview roles organizations their support programs. It examines key partnerships such research expertise Coalition Epidemic Preparedness Innovations (CEPI), control supply chain Global Alliance Vaccines Immunization (GAVI), transfer capabilities International Vaccine Institute (IVI) supporting development, production, technologies priority announced WHO CEPI programs policies identify effective rapidly caused emerging diseases. Methods: focused on crisis. Literature data was collected, compared, analyzed national literature search sites, referring articles, journals, reports, publications, books, guidelines, clinical trial data, related reports. addition, websites WHO, CEPI, GAVI, IVI, were used examine projects, initiatives, collaborations reviews case methods. Results: COVID-19 brought focus necessity developing innovative platforms. Despite initial concerns, swift integration cutting-edge technologies, mass production capabilities, collaboration made messenger RNA (mRNA) vaccines game-changing technology. As result successful application novel platforms, it is important address remaining challenges, safety equitable distribution. To achieve this, essential review regulatory, policy, initiatives that implemented response pandemic, with particular emphasis stages distribution, prepare pandemics. An analysis implies balanced development. Also, IVI play preparedness preventive These collaborated improve accessibility vaccines, strengthen diseases, issues. demonstrates how synergistic WHO’s standardized CEPI’s expertise, GAVI’s IVI’s can be united create process Conclusions: preparation pandemics, essential. should include investment both viral vector-based traditional goal develop applied efficiently increase manufacturing Moreover, setting direction networking preparing limitations previous responses. result, transforming threats from unpredictable surmountable expected systems reduce social economic burden long term.
Язык: Английский
Процитировано
1
Advanced Materials, Год журнала: 2023, Номер 36(13)
Опубликована: Ноя. 6, 2023
Abstract Messenger RNA (mRNA)‐based therapy has emerged as a powerful, safe, and rapidly scalable therapeutic approach that involves technologies for both mRNA itself the delivery vehicle. Although there are some unique challenges different applications of therapy, common challenge all therapeutics is transport into target cell cytoplasm sufficient protein expression. This review focused on behaviors at cellular level nanotechnology‐mediated systems, which have not been comprehensively reviewed yet. First, four main introduced, including immunotherapy, replacement genome editing, reprogramming. Second, types cargos systems summarized. Third, strategies to enhance efficiency during trafficking process highlighted, accumulation cell, internalization endosomal escape, release from nanocarrier, translation protein. Finally, opportunities development presented. can provide new insights future fabrication nanocarriers with desirable performance.
Язык: Английский
Процитировано
17
Advanced Science, Год журнала: 2023, Номер 10(35)
Опубликована: Окт. 31, 2023
Abstract Chimeric antigen receptor T‐cell (CAR‐T) therapy has emerged as a highly efficacious treatment modality for refractory and relapsed hematopoietic malignancies in recent years. Furthermore, CAR technologies cancer immunotherapy have expanded from CAR‐T to CAR‐natural killer cell (CAR‐NK), CAR‐cytokine‐induced (CAR‐CIK), CAR‐macrophage (CAR‐MΦ) therapy. Nevertheless, the high cost complex manufacturing processes of ex vivo generation autologous products hampered broader application. There is an urgent need develop efficient economical paradigm shift exploring new sourcing strategies engineering approaches toward generating CAR‐engineered immune cells benefit patients. Currently, researchers are actively investigating various optimize preparation these potent immunotherapeutic agents. In this work, latest research progress summarized. Perspectives on future provided, approaches, diverse sources used their development focused upon.
Язык: Английский
Процитировано
16
Materials Today Bio, Год журнала: 2024, Номер 26, С. 101101 - 101101
Опубликована: Май 29, 2024
Messenger ribonucleic acid (mRNA) has emerged as a promising molecular preventive and therapeutic approach that opens new avenues for healthcare. Although the use of delivery systems, especially lipid nanoparticles (LNPs), greatly improves efficiency stability mRNA, mRNA tends to accumulate in liver hardly penetrates physiological barriers reach target site after intravenous injection. Hence, rational design targeting strategies aimed at directing specific tissues cells remains an enormous challenge therapy. High-throughput screening (HTS) is cutting-edge targeted technique capable synthesizing chemical compound libraries large-scale experiments validate system. In this review, we firstly provide overview conventional low-throughput strategies. Then latest advancements HTS techniques delivery, encompassing optimizing structures vehicles developing surface ligands, well applications extrahepatic systemic diseases are comprehensively summarized. Moreover, illustrate selection administration routes delivery. Finally, challenges field potential solutions tackle them proposed, offering insights future development toward
Язык: Английский
Процитировано
5
Molecules, Год журнала: 2024, Номер 29(11), С. 2461 - 2461
Опубликована: Май 23, 2024
mRNA vaccines are entering a period of rapid development. However, their synthesis is still plagued by challenges related to impurities and fragments (incomplete mRNA). Most products transcribed in vitro fragments. Only full-length transcripts containing both 5′-cap 3′-poly(A) structure viable for vivo expression. Therefore, RNA the primary product-related that significantly hinder efficacy must be effectively controlled; these species believed originate from either hydrolysis or premature transcriptional termination. In manufacturing commercial vaccines, T7 polymerase-catalyzed transcription (IVT) well-established method synthesizing long transcripts. This study identified pivotal domain on polymerase associated with erroneous release. By leveraging advantageous properties mutant precisely optimized IVT process parameters, we successfully achieved an integrity exceeding 91%, thereby further unlocking immense potential therapeutics.
Язык: Английский
Процитировано
4
Journal of Pharmaceutical and Biomedical Analysis, Год журнала: 2024, Номер 249, С. 116352 - 116352
Опубликована: Июль 14, 2024
Messenger RNA (mRNA) is rapidly growing as a therapeutic modality for vaccination and the treatment of wide range diseases. As result, there an increased demand mRNA-based analytical methods capable assessing purity stability, which are considered critical quality attributes (CQAs). In recent decades capillary electrophoresis (CE) has emerged alongside liquid chromatography (LC) important tool assessment stability mRNA therapeutics. CE offers variety advantages over conventional LC or gel-based methods, including reduced injection volume, resolution, separation efficiency. this study we compared CE-based methods: Agilent 6000 Nano Kit, Revvity Reagent Sciex 9000 Purity Integrity HS Kit. These were evaluated on their vendor-recommended instruments: Bioanalyzer, LabChip GXII, PA800 Plus, Fragment Analyzer, respectively. We assessed ability these to measure integrity, purity, stability. Furthermore, several parameters each method also assessed: selectivity, precision, analysis time, ease use. Based our results, all four suitable use in characterization vitro transcribed (IVT) mRNA, depending intended application. The kit achieved highest selectivity resolving power with other making it most high-resolution, in-depth sample characterization. comparison, Kit lower but faster times make them more high-throughput screening applications.
Язык: Английский
Процитировано
4
Small Science, Год журнала: 2024, Номер unknown
Опубликована: Июль 30, 2024
Lipid nanoparticles and polymeric are promising biomaterial platforms for robust intracellular DNA mRNA delivery, highlighted by the widespread use of nanoparticle‐ (NP) based vaccines to help end COVID‐19 pandemic. Recent research has sought adapt this nanotechnology transfect engineer immune cells in vivo. The system is an especially appealing target due its involvement many different diseases, ex vivo‐engineered cell therapies like chimeric antigen receptor (CAR) T therapy have already demonstrated remarkable clinical success certain blood cancers. Although gene delivery can potentially address some cost manufacturing concerns associated with current autologous therapies, transfecting vivo challenging. Not only extrahepatic NP lymphoid organs difficult, but particular resistance transfection. Despite these challenges, modular nature NPs allows researchers examine critical structure–function relationships between a particle's properties ability specifically Herein, several nanomaterial components outlined, including targeting ligands, nucleic acid cargo, chemical properties, physical route administration optimal
Язык: Английский
Процитировано
4