Drug Database
FA

Factor IX (AlphaNine SD / AlphaNine)

✓ Approved

Mitsubishi Tanabe Pharma Corporation · F9 · 细胞治疗

什么是 Factor IX?

Factor IX 是一种细胞治疗,由Mitsubishi Tanabe Pharma Corporation研发。该药已获批,用于治疗相关适应症,给药途径:Injectable (Others)、Intravenous (IV)。

药物档案

商品名AlphaNine SD, AlphaNine
公司Mitsubishi Tanabe Pharma Corporation
药物类别细胞治疗
分子靶点F9
给药途径Injectable (Others), Intravenous (IV)
状态Approved

作用机制

分子靶点

Factor IX 作用于 1 个分子靶点:

F9coagulation factor IX (P19, F9 p22)
需要更深入的分析?Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

Factor IX 针对 3 个适应症,涉及 2 个治疗领域。

治疗领域疾病/病症分期
Vascular disordersExtravasation blood✓ Approved
Congenital, familial and genetic disordersFactor IX deficiency✓ Approved
Vascular disordersHaemorrhage✓ Approved

相关研究文献

PubMedRSC advances2026-09-10

Harnessing coumarin privilege to achieve potency and selectivity: hypoxia-targeted alkyl coumarin-tethered phenyl quinazolinones as dual hCA IX/HSP90α inhibitors with chemosensitizing activity.

Elgohary Amal S AS, El-Hamamsy Mervat H MH, Sharafeldin Nabaweya N, Giovannuzzi Simone S et al.

Hypoxic tumor regions remain challenging to treat in breast cancer therapy due to extracellular acidification, chemoresistance, and other adaptive mechanisms promoted by the coordinated actions of tumor-associated carbonic anhydrase IX (hCA IX), heat shock protein 90 alpha (HSP90α), and other proteins. Inspired by the privileged role of coumarins as selective hCA IX inhibitors and as HSP90α modulators, together with the ATP-mimetic and anticancer properties of phenyl quinazolinones, a series of alkyl coumarin-tethered phenyl quinazolinones (4a-l) were rationally designed and synthesized as hypoxia-targeted dual hCA IX/HSP90α inhibitors. 1H NMR, 13C NMR-DEPTQ, HRMS, elemental analysis, and HPLC unequivocally established the structures of the synthesized compounds. The synthesized hybrids exhibited potent, highly selective inhibition of hCA IX (in the nanomolar range), while displaying negligible activity against the off-target isoforms hCA I and II. Compound 4b emerged as the lead derivative, exhibiting a K I value of 51.7 nM against hCA IX together with exceptional selectivity (>1934-fold over hCA I and hCA II). Remarkably, 4b also exhibited potent HSP90α inhibitory activity (IC50 = 13.21 nM), comparable to that of ganetespib. Furthermore, 4b preferentially suppressed the proliferation of hypoxic MCF-7 and MDA-MB-231 breast cancer cells, sensitized them to doxorubicin, and induced p53-mediated mitochondrial apoptosis. Molecular docking and molecular dynamics simulations against hCA IX and HSP90α, along with in silico ADME and toxicity studies, further supported the favorable binding behavior, stability, and druglike characteristics of the lead compound.

PMID 42719674
阅读全文 →
PubMedFrontiers in pharmacology2026-09-10

Biologic and targeted synthetic therapies in ankylosing spondylitis: a pharmacological review.

Ameer Omar Z OZ, Temsah Reem R, Alsouss Yara O YO, Al-Amoudi Raghad R et al.

Ankylosing spondylitis (AS) is a prototypical axial spondyloarthritis (axSpA) defined by chronic inflammatory back pain, progressive spinal stiffness, and frequent extra-articular manifestations such as acute anterior uveitis (21%-33%), inflammatory bowel disease (5%-10%), and psoriasis (4%-13%). Management has shifted from non-steroidal anti-inflammatory drugs (NSAIDs) to highly targeted biologic and synthetic disease-modifying antirheumatic drugs (DMARDs). This comprehensive review focuses on the current evidence for biologic and targeted synthetic therapeutics in the management of AS, emphasizing mechanisms of action, regulatory approvals, dosing regimens, and clinical efficacy. Three major therapeutic classes are identified: two biologic DMARD classes, comprising tumor necrosis factor-alpha (TNF-α) inhibitors and interleukin-17 (IL-17) inhibitors, and the targeted synthetic DMARDs, namely, the Janus kinase-signal transducer and activator of transcription (JAK-STAT) inhibitors. TNF inhibitors (adalimumab, infliximab, etanercept, golimumab, certolizumab pegol) typically achieve Assessment of Spondyloarthritis International Society 40% improvement (ASAS40) rates of 40%-58% at 12-24 weeks versus 13%-24% with placebo, and maintain clinical benefit in many patients over 5-8 years. IL-17 inhibitors (secukinumab, ixekizumab, bimekizumab, brodalumab, netakimab) yield broadly similar ASAS40 responses (30%-50% versus 10%-20% placebo) and are particularly useful in patients with concomitant psoriasis with substantially reduced risk of tuberculosis reactivation. JAK inhibitors (tofacitinib, upadacitinib) represent the newest class, offering oral administration with ASAS40 responses of 40%-52% versus 13%-26% for placebo in both biologic-naïve and biologic-experienced AS, though cardiovascular safety considerations necessitate careful patient selection. Therapeutic selection should consider comorbidities (uveitis favors TNF monoclonal antibodies; psoriasis favors IL-17 inhibitors), safety profile, route of administration, and sequencing after non-steroidal anti-inflammatory drug failure. Understanding these pharmacological distinctions enables personalized therapeutic decisions based on the specific molecular pathology and risk profile of the individual patient.

PMID 42719014
阅读全文 →
PubMedBMC pediatrics2026-09-10

Psychometric properties of the Turkish version of the Preschool Eating, Lifestyle, and Sleeping Attitudes (PRELSA) scale.

Bolacali Edanur Tar ET, Bolacali Memiş M

Childhood obesity in preschool years is strongly influenced by modifiable parental attitudes and behaviors. This study examined the validity and reliability of the Turkish version of the Preschool Eating, Lifestyle, and Sleeping Attitudes (PRELSA) Scale, which assesses parental attitudes related to childhood obesity during the preschool period. This methodological study was conducted with 460 parents of children aged 2-6 years from seven preschools in Central Anatolia, Türkiye. Data were collected using a family and child information form and the PRELSA Scale. The adaptation process included forward-backward translation, expert review, face validity assessment, and psychometric evaluation. Data were analyzed using exploratory and confirmatory factor analyses, Cronbach's alpha, test-retest reliability, and intraclass correlation coefficients (ICC). The Turkish version comprised 39 items across 13 subscales. The overall Cronbach's alpha coefficient was 0.887. Exploratory factor analysis explained 58.5% of the total variance, with factor loadings ranging from 0.353 to 0.890. Confirmatory factor analysis indicated acceptable model fit (χ²/df = 1.434, RMSEA = 0.031, SRMR = 0.046, CFI = 0.904, IFI = 0.907, GFI = 0.911, TLI = 0.888). Test-retest reliability was high (r = 0.911), and the ICC was 0.865 (95% CI: 0.738-0.933), indicating good temporal stability. The Turkish PRELSA Scale is a valid and reliable instrument for assessing parental attitudes toward eating behaviors, physical activity, screen use, and sleep routines in early childhood.

PMID 42717323
阅读全文 →
PubMedThe AAPS journal2026-09-10

Immunogenicity Assays Used in Support of Marketing Applications of Oligonucleotide Therapeutics.

Arfvidsson Cecilia C, Chen Lin-Zhi LZ, Mohapatra Susovan S, Mukherjee Kamalika K et al.

Oligonucleotide therapeutics (ONT) continue to emerge as a versatile new class of medicines. While relatively small in terms of molecular weight, ONT may trigger formation of anti-drug antibodies (ADA), which in turn may impact pharmacokinetics, pharmacodynamics, efficacy, and safety. Bioanalytical methodology for detection of ADA against protein-based therapeutics is well established, however, detection of ADA against ONT presents its own unique challenges. In this manuscript, the Immunogenicity Sub-team of AAPS Bioanalytical Community Oligonucleotide Discussion Group, reviewed bioanalytical assays used to detect and characterize ADA as reported in documents supporting marketing applications of oligonucleotide therapeutics. Based on the available data and the Authors' own experience, a direct ELISA is by far the most common format used for detection of ADA against ONT. Characterization of ADA neutralizing activity is not performed. No issues with insufficient drug tolerance have been reported and postmarketing requirements to improve assay sensitivity are rare. Data generated in the Authors' laboratories indicate that difficulties with low sensitivity of immunogenicity assays for ONT appear to stem from difficulties in raising and purifying high affinity positive control antibodies. Given the inherently low immunogenic potential of ONTs, generation of positive controls with sufficient affinity and titer may necessitate multiple immunizations with immunogens incorporating repetitive sequence motifs, together with alternation of carrier proteins across immunization cycles.

PMID 42717178
阅读全文 →
PubMedRSC advances2026-09-10

4D nanoimaging-guided smart therapeutics: spatiotemporal control of disease microenvironments.

Sarma Arnabjyoti Deva AD, Devi Moitrayee M, Webster Thomas J TJ, Kumar Dinesh D et al.

A revolutionary shift in the field of nanotheranostics is the development of smart therapeutics guided by 4D nanoimaging, which addresses the shortcomings of traditional platforms that rely on static imaging and passive drug delivery. The spatial and temporal control of these systems can be accurately tuned to modulate dynamics in disease microenvironment assays, especially for complex diseases, such as cancer and inflammatory diseases. Recent developments in stimulus-responsive nanomaterials and real-time visualization modalities have enabled more specific and less damaging adaptive, feedback-based therapeutic interventions. This review presents an overview of the recent literature on the design, operating principles, and biomedical applications of 4D nanoimaging-guided smart therapeutics. Particular attention is paid to building nanoplatforms that are responsive to endogenous and exogenous signals and advanced imaging tools (e.g., near-infrared fluorescence and photoacoustic imaging), as well as integrating them with artificial intelligence to establish predictive and closed-loop therapeutic platforms. Applicable literature has been critically reviewed with regards to the performance parameters of spatial-temporal resolution, therapy release performance, and therapeutic effects. The reviewed literature shows how the 4D nanoimaging platforms reach high spatiotemporal precision to allow real-time monitoring of the biodistribution of nanoparticles and disease progression. The controlled drug release efficiencies of the stimuli-responsive systems typically range from 70% to 90%, and the integration of multimodal imaging provides better diagnostic performance and tissue penetration. The closed-loop therapeutic regulation that this platform provides through its integration with AI models and a biosensing interface dramatically enhances nanomedicine targeting specificity and therapeutic indices over conventional approaches. A complete paradigm shift to adaptive, personalized smart therapeutics directed by the 4D nanoimaging system will facilitate the development of real-time, microenvironment-specific interventions. While preclinical data have been encouraging, issues like biocompatibility, scalability, and clinical translation remain. It is expected that the integration of AI-driven analytics in multifunctional nanoplatforms will jumpstart the translation of these technologies into clinical settings.

PMID 42719203
阅读全文 →
PubMedPharmacogenomics2026-09-10

Development and psychometric validation of the pharmacogenetic Knowledge, attitudes, and Awareness scale (P-KAAS): an initial validation study in undergraduate nursing students.

Yesilot Sukriye S, Ceylan Hatice H, Kosar Sahin Cansu C, Aydin Acar Cigdem C

Pharmacogenetics is a key component of precision medicine, yet its integration into clinical practice remains limited. This study aimed to develop and validate the Pharmacogenetic Knowledge, Attitudes and Awareness Scale (P-KAAS) and assess nursing students' pharmacogenetic competencies. This methodological, cross-sectional scale-development study included 312 undergraduate nursing students. Content validity was evaluated by an expert panel using the Davis technique. Construct validity was assessed using exploratory factor analysis (EFA) and known-groups validity analyses. Internal consistency, item-total correlations, and test-retest reliability were examined. Students demonstrated moderate awareness and positive attitudes toward pharmacogenetics but limited detailed knowledge. EFA identified a two-factor structure explaining 60.99% of the total variance, with factor loadings ranging from 0.438 to 0.725. Known-groups validity analyses demonstrated significant differences in P-KAAS scores according to academic year and prior exposure to pharmacogenetics. Cronbach's alpha was 0.929 for the total scale, 0.696 for the Knowledge subscale, and 0.938 for the Attitude and Awareness subscale. Test-retest correlations ranged from 0.87 to 0.90. The P-KAAS demonstrated satisfactory psychometric properties among undergraduate nursing students. Further validation in other healthcare professional groups is warranted before broader application.

PMID 42719992
阅读全文 →

注册免费账户还可查看另外 9996 篇文献

免费注册查看全部文献 →

了解更多Factor IX