Expert Opinion on Emerging Drugs最新文献_第2页

At the forefront of gene-based therapies in Dravet syndrome. 在基因治疗的最前沿。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-09-01 Epub Date: 2026-07-08 DOI: 10.1080/14728214.2026.2683554

Antonella Riva, Sara Di Gioacchino, Greta Volpedo, Beatrice Risso, Antonella De Lillo, Federico Zara, Antonio Falace, Pasquale Striano

{"title":"At the forefront of gene-based therapies in Dravet syndrome.","authors":"Antonella Riva, Sara Di Gioacchino, Greta Volpedo, Beatrice Risso, Antonella De Lillo, Federico Zara, Antonio Falace, Pasquale Striano","doi":"10.1080/14728214.2026.2683554","DOIUrl":"10.1080/14728214.2026.2683554","url":null,"abstract":"Introduction: Dravet syndrome (DS), a catastrophic developmental and epileptic encephalopathy primarily caused by SCN1A haploinsufficiency, remains largely refractory to current antiseizure medications. In recent years, gene-targeted therapies, including antisense oligonucleotides (ASOs)-and viral-based therapies, have emerged as promising disease-modifying strategies.Areas covered: Preclinical in vitro and in vivo models demonstrated that ASO-mediated modulation of SCN1A can restore transcript and protein levels, normalize interneuron excitability, and extend survival. Key advances include the discovery of deep-intronic 'poison' exons expanding actionable ASO targets and the demonstration of long-term functional rescue after early-life administration. Complementary strategies, such as SCN8A modulation, SCN1A viral delivery, and tau suppression, further broadened therapeutic options. Clinically, the ASO STK-001 trial showed favorable safety, pharmacodynamic activity, and durable seizure reduction in Phase 1/2a and open-label extension trials, alongside improvements in adaptive behavior and cognition.Expert opinion: ASO-based modulation of SCN1A represents a pivotal advance in DS therapy, translating robust preclinical efficacy into early clinical benefit. Optimization of dosing regimens, durability, and combinatorial approaches targeting parallel molecular pathways will be critical to realize precision gene therapy for DS. Viral gene therapy approaches constitute a promising therapeutic platform, but comprehensive validation will be essential to ascertain their immunogenic safety and demonstrate clinical efficacy.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"95-105"},"PeriodicalIF":3.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148210802","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Emerging treatment options for chronic obstructive pulmonary disease: a look into 2026 and beyond. 慢性阻塞性肺疾病的新兴治疗方案：展望2026年及以后。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-09-01 Epub Date: 2026-06-22 DOI: 10.1080/14728214.2026.2688171

Carolyn J Wang, Clarus Leung, Don D Sin

{"title":"Emerging treatment options for chronic obstructive pulmonary disease: a look into 2026 and beyond.","authors":"Carolyn J Wang, Clarus Leung, Don D Sin","doi":"10.1080/14728214.2026.2688171","DOIUrl":"10.1080/14728214.2026.2688171","url":null,"abstract":"Introduction: As a leading cause of worldwide mortality, chronic obstructive pulmonary disease (COPD) is progressive and irreversible, leading to significant challenges with disease management and treatment. This is further complicated by the heterogenous nature of COPD, which is represented by the multiple endotypes that have variable responses to available treatments, thereby negating the one-size-fits-all approach. While recent clinical studies describe the effectiveness of existing therapies, further investigation is required to align COPD endotypes with the appropriate treatment options.Areas covered: In this review, we discuss the known biomarkers pertaining to both direct and indirect measures of the inflammatory milieu and structural abnormalities in COPD. Combined use of conventional anti-inflammatory and bronchodilator treatments provide some symptomatic relief by reducing exacerbation frequency, however, for some patients, long-term corticosteroid use can increase the risk developing pneumonia and subsequent mortality. Biologics are at the forefront of emerging therapies for COPD, which shifts the target from widespread airway inflammation to pinpoint precise inflammatory markers involved in pathophysiological mechanisms of COPD.Expert opinion: Identifying reliable airway-derived biomarkers in COPD will drive the development of therapies that can facilitate a precision medicine approach for patients with COPD.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"123-136"},"PeriodicalIF":3.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148276799","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Emerging biological drugs for the treatment of patients with metastatic HER2+ gastroesophageal adenocarcinomas. 新兴生物药物治疗转移性HER2+胃食管腺癌。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-09-01 Epub Date: 2026-07-20 DOI: 10.1080/14728214.2026.2705211

Jane E Rogers, Ashley Horak, Alicia Kroll, Jaffer A Ajani

{"title":"Emerging biological drugs for the treatment of patients with metastatic HER2+ gastroesophageal adenocarcinomas.","authors":"Jane E Rogers, Ashley Horak, Alicia Kroll, Jaffer A Ajani","doi":"10.1080/14728214.2026.2705211","DOIUrl":"10.1080/14728214.2026.2705211","url":null,"abstract":"Introduction: Advanced gastroesophageal adenocarcinomas (GEAs) continue to represent an area in need of new drug development. Targeting human epidermal growth factor receptor-2 (HER2) in GEAs has been standard since 2010 and continues to be an area of further development.Areas covered: Trastuzumab became the first targeted agent approved for GEA. Added to front-line fluoropyrimidine plus platinum chemotherapy in HER2+ metastatic disease, trastuzumab improved survival. Pembrolizumab was approved in HER2 positive GEA for those PD-L1 positive with modest benefit. Since trastuzumab's approval, other anti-HER2 agents had limited impact until 2021 with the FDA approval of trastuzumab deruxtecan. Trastuzumab deruxtecan represents an option after trastuzumab-based therapy fails. Other agents are currently being investigated.Expert opinion: With the abundance of anti-HER2 therapies in the investigative pipeline, understanding key resistance mechanisms seen in HER2+ GEA will be the path to success. Technology advancements to provide real‑time precision medicine are likely in the coming years. We hope these advancements can help combat and/or understand some of the intratumoral heterogeneity, HER2 expression changes, and tumor microenvironment resistance pathways seen in HER2+ GEA.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"145-153"},"PeriodicalIF":3.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148445269","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Beyond triptans in menstrual migraine: the emerging role of CGRP-targeted therapies. 除了曲坦类药物在经期偏头痛中的作用：cgrp靶向治疗的新作用。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-09-01 Epub Date: 2026-06-16 DOI: 10.1080/14728214.2026.2690195

Lanfranco Pellesi, Damiana Scuteri, Andrea Burgalassi, Alberto Chiarugi, Paolo Martelletti

{"title":"Beyond triptans in menstrual migraine: the emerging role of CGRP-targeted therapies.","authors":"Lanfranco Pellesi, Damiana Scuteri, Andrea Burgalassi, Alberto Chiarugi, Paolo Martelletti","doi":"10.1080/14728214.2026.2690195","DOIUrl":"10.1080/14728214.2026.2690195","url":null,"abstract":"Introduction: Menstrual migraine is a common and disabling subtype of migraine, linked to hormonal fluctuations and characterized by attacks that are often more severe, longer lasting and less responsive to treatment than non-menstrual episodes. Despite its relevance, menstrual migraine remains underdiagnosed and undertreated.Areas covered: This review examines the role of therapies targeting the calcitonin gene-related peptide (CGRP) pathway in menstrual migraine. We summarize current evidence on monoclonal antibodies and gepants, focusing on their efficacy in hormonally triggered attacks and their potential advantages over traditional treatments.Expert opinion: CGRP-targeted therapies represent a promising mechanism-based approach for menstrual migraine, although their role is not yet fully defined. These treatments reduce overall migraine burden, but the perimenstrual component may remain relatively resistant. The lack of dedicated clinical trials is a major limitation. Among available options, gepants offer the greatest potential due to their flexibility for both acute and short-term preventive use. Future research should focus on time-specific treatment strategies and a broader understanding of the underlying biology, including the role of additional neuropeptides.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"137-144"},"PeriodicalIF":3.8,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148249978","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Claudin-6 as a potential target in the treatment of ovarian cancer: a review of emerging drugs. Claudin-6作为卵巢癌治疗的潜在靶点：新兴药物综述

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-07-26 DOI: 10.1080/14728214.2026.2706609

Sarah Ottum, Namrata Sethi, Cem Demirkiran, Orazio De Tommasi, Alessandro D Santin

{"title":"Claudin-6 as a potential target in the treatment of ovarian cancer: a review of emerging drugs.","authors":"Sarah Ottum, Namrata Sethi, Cem Demirkiran, Orazio De Tommasi, Alessandro D Santin","doi":"10.1080/14728214.2026.2706609","DOIUrl":"10.1080/14728214.2026.2706609","url":null,"abstract":"Introduction: Claudins are integral components of tight junctions with emerging roles in cancer biology. Dysregulated claudin expression during malignant transformation exposes these proteins on the tumor cell surface, creating opportunities for targeted therapy. Claudin-6, an oncofetal protein largely absent from normal adult tissues, is aberrantly expressed in a significant subset of ovarian cancers, making it an attractive therapeutic target.Areas covered: This review summarizes the biological rationale for targeting claudin-6 in ovarian cancer and evaluates current therapeutic strategies, including monoclonal antibodies, antibody-drug conjugates, bispecific T-cell engagers, CAR-T cells, and CAR-NK cells. The analysis is based on a structured literature search conducted in major biomedical databases, including MEDLINE (via PubMed), EMBASE, Scopus, and Web of Science, as well as ClinicalTrials.gov for ongoing studies.Expert opinion: Claudin-6-targeted therapies represent a promising advancement for patients with recurrent or platinum-resistant ovarian cancer. Among current approaches, ADCs appear closest to broad clinical applicability due to their potency and manageable safety profiles. Future success will depend on standardized diagnostic assays, biomarker-driven trial designs, and rational combination strategies to overcome resistance and maximize durable clinical benefit.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"1-8"},"PeriodicalIF":3.8,"publicationDate":"2026-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148469869","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Drug discovery progress in targeting the renin-angiotensin-aldosterone system for heart failure. 针对肾素-血管紧张素-醛固酮系统治疗心力衰竭的药物发现进展。

IF 3.8 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-07-21 DOI: 10.1080/14728214.2026.2706607

Diksha Choudhary, Nisha Bansal, Bhupinder Kumar

引用次数: 0

Emerging microbiome-based therapies for atopic dermatitis: clinical insights and future development. 新兴的基于微生物组的治疗特应性湿疹：临床见解和未来发展。

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-05-06 DOI: 10.1080/14728214.2026.2667256

Kingsley Osei-Karikari, Kyle Jones, Anna Erickson, Ian A Myles

{"title":"Emerging microbiome-based therapies for atopic dermatitis: clinical insights and future development.","authors":"Kingsley Osei-Karikari, Kyle Jones, Anna Erickson, Ian A Myles","doi":"10.1080/14728214.2026.2667256","DOIUrl":"10.1080/14728214.2026.2667256","url":null,"abstract":"Introduction: Despite growing evidence that environmental factors and microbiome dysbiosis constitute the majority of disease pathogenesis, most current therapies target inflammation, barrier dysfunction, and/or S. aureus overgrowth. However, given the emerging understanding of microbiome-mediated and environmentally driven disease mechanisms, expanding therapeutic strategies to include dysbiosis and upstream environmental contributors represents a promising direction for future research.Areas covered: This manuscript will review the strengths and limitations of the standard treatments for AD, including emollients, topical corticosteroids, calcineurin inhibitors, and systemic immunomodulators. The focus, however, will be on the emerging microbiome-based therapies, specifically products containing live microorganisms (biotherapeutics). The potential for, and challenges against, biotherapeutics to grow within the market of AD treatment will be discussed.Expert opinion: Immune suppressive approaches will remain limited to symptomatic control. These treatments will also be limited by tradeoffs inherent to the balance between symptom control and side effects of immune suppression. To successfully aide patients, topical biotherapeutics will need to overcome pharmaceutical-centric paradigms, academic dogma, and regulatory inertia. Limitations in current therapies and patient needs, along with the potential to offer prevention against AD development, will likely propel biotherapeutics forward.","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":" ","pages":"17-28"},"PeriodicalIF":2.7,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147766804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Phase II and III trials in systemic sclerosis: from recent FDA approvals to emerging applications of cellular therapies. 系统性硬化症的II期和III期试验：从最近FDA批准到细胞疗法的新应用。

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-05-18 DOI: 10.1080/14728214.2026.2675269

Alain Lescoat, Eisha Naveed, Dinesh Khanna

引用次数: 0

Emerging pharmacotherapies in Gaucher disease. 戈谢病的新兴药物疗法。

IF 2.7 3区医学

Expert Opinion on Emerging Drugs Pub Date : 2026-03-01 Epub Date: 2026-05-19 DOI: 10.1080/14728214.2026.2675279

Gregory M Pastores

引用次数: 0

Injectable, oral, gene-based, and vaccine-driven PCSK9-targeted therapies: emerging clinical insights shaping the future of hypercholesterolemia management. 注射、口服、基于基因和疫苗驱动的pcsk9靶向治疗：塑造高胆固醇血症管理未来的新兴临床见解