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Factor X + Factor IX (Factor X P Behring)

✓ Approved

CSL Behring · F9 · 细胞治疗

什么是 Factor X + Factor IX？

Factor X + Factor IX 是一种细胞治疗，由CSL Behring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Factor X P Behring
公司CSL Behring
药物类别细胞治疗
分子靶点F9, F10
给药途径Injectable (Others), Intravenous (IV)
状态Approved
来源： ClinicalTrials.gov, CSL Behring

作用机制

分子靶点

Factor X + Factor IX 作用于 2 个分子靶点：

F9coagulation factor IX (P19, F9 p22)
F10coagulation factor X (FXA, FX)
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 X + Factor IX 针对 2 个适应症，涉及 1 个治疗领域。

治疗领域疾病/病症分期
Congenital, familial and genetic disordersFactor IX deficiency✓ Approved
Congenital, familial and genetic disordersFactor X deficiency✓ Approved

相关研究文献

PubMedInternational journal of molecular sciences2026-10-27

Fidanacogene Elaparvovec Gene Therapy: Budesonide Treatment of a Late Decline in Factor IX Expression in a Patient with Hemophilia B.

Herbst Kerstin K, Pezeshkpoor Behnaz B, Klein Claudia C, Albert Thilo T et al.

Fidanacogene elaparvovec is an adeno-associated virus (AAV)-based gene therapy that enables endogenous factor IX (FIX) expression in hemophilia B, potentially eliminating the need for prophylactic FIX replacement. Post-treatment monitoring focuses on FIX durability, hepatic safety, and immune-mediated loss of expression. A 40-year-old man with severe hemophilia B received a single intravenous dose of fidanacogene elaparvovec in the phase 3 BENEGENE-2 trial (NCT03861273). FIX activity was assessed by one-stage and chromogenic assays. Liver enzymes, clinical parameters, and adverse events were monitored. Prednisolone was initiated for liver enzyme elevation and later switched to budesonide in response to declining FIX levels. Follow-up continued for 4 years. FIX activity peaked at week 6 (~30 IU/dL) and remained stable until week 16. Moderate ALT elevation (peak ~70 U/L) responded to prednisolone. Between weeks 22-28, FIX levels declined without ALT elevation. Budesonide (9 mg/day) was started at week 25 for 3 months and gradually tapered, stabilizing FIX activity at 10-20 IU/dL. This level has been maintained for 4 years without bleeding episodes or FIX replacement. Fidanacogene elaparvovec provided sustained, clinically meaningful FIX expression. Budesonide may offer an effective hepatocyte-targeted alternative to systemic corticosteroids while supporting long-term transgene expression.

PMID 42941822
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PubMedBiomedicines2026-10-27

Histopathological Remodeling and Reduced Hypoxia-Related Marker Expression Following Marsupialization of Ameloblastoma: A Paired Analysis.

Ishida Shuichi S, Takabatake Kiyofumi K, Arashima Takuma T, Funahashi Hiroshi H et al.

Background: Marsupialization is widely used as a jaw-preserving treatment for large cystic ameloblastomas and is generally believed to reduce tumor volume through decompression. However, the biological mechanisms underlying tumor regression remain poorly understood. This study aimed to investigate the histopathological and hypoxia-related molecular changes in ameloblastoma before and after marsupialization using paired specimens from the same patients. Methods: This retrospective paired study included 13 patients with ameloblastoma who underwent marsupialization (median duration, 3 months; range, 1-12 months) followed by enucleation with peripheral ostectomy. Histopathological and immunohistochemical changes were compared between paired pre- and post-marsupialization specimens obtained from the same patients. Expression of the hypoxia-related molecules HIF-1α, carbonic anhydrase IX (CA IX), glucose transporter 1 (GLUT1), vascular endothelial growth factor (VEGF), and the proliferation marker Ki-67 was evaluated. Results: Marsupialization was associated with characteristic histopathological alterations, including increased squamous metaplasia, reduced stellate reticulum-like morphology, and a shift from myxoid to fibrous stromal patterns. Nuclear HIF-1α expression decreased significantly after marsupialization, accompanied by coordinated reductions in CA IX, GLUT1, VEGF, and the Ki-67 labeling index. These findings were consistent with coordinated attenuation of hypoxia-associated signaling together with reduced Ki-67 labeling. Conclusions: This study provides within-patient pathological evidence of histopathological remodeling and reduced hypoxia-related marker expression following marsupialization in ameloblastoma. These findings suggest that the tissue response to marsupialization may involve attenuation of hypoxia-associated signaling.

PMID 42940142
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PubMedNature communications2026-10-27

Efficient transport kinetics of indirect excitons in van der Waals heterostructure.

Zhou Zhiwen Z, Brunner W J WJ, Szwed E A EA, Henstridge H H et al.

Exciton transport is fundamental for understanding transport phenomena in bosonic systems and for exploring excitation energy transfer in materials. Spatially indirect excitons (IXs) have long lifetimes allowing them to form quantum bosonic states and travel long distances. Van der Waals heterostructures form a new materials platform for exploring IX transport. Disordered in-plane potentials suppress IX transport due to IX localization and scattering. In this work, we found the efficient IX transport kinetics characterized by anomalously high IX mobility. The efficient IX transport kinetics is observed in the presence of substantial in-plane disorder. The anomalously high IX mobility, its disappearance with increasing temperature, and its non-monotonic dependence on density are qualitatively consistent with theoretical predictions of IX superfluidity.

PMID 42934215
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PubMedMolecules (Basel, Switzerland)2026-10-27

Design, Synthesis, and Biological Evaluation of Novel Dithiocarbamate-Based Human Carbonic Anhydrase IX/XII Inhibitors as Potential Anti-Glioblastoma Agents.

Altıntop Mehlika Dilek MD, Nadaroglu Hayrunnisa H, Yesilyurt Fatma F, Yılmaz Savaş Nalan N et al.

To develop human carbonic anhydrase (hCA) IX/XII-targeted therapeutics for glioblastoma multiforme (GBM), novel dithiocarbamate (DTC)-based compounds were synthesized and evaluated for their inhibitory activity against hCA I, II, IX, and XII isoforms. The most potent hCA IX/XII inhibitors were further investigated for their cytotoxic activity against U87 and T98 glioblastoma cells, selectivity toward healthy neuronal cells, and their effects on oxidative stress, inflammatory mediators, and cell death-related pathways. Compounds 4, 9, and 30 emerged as the most promising candidates, with compounds 9 and 30 exhibiting potent cytotoxic activity against both U87 and T98 cells. These compounds acted as dual hCA IX/XII inhibitors with a clear preference for hCA XII. Compounds 9 and 30 modulated the oxidative stress-inflammation axis through regulation of cellular redox balance, and inflammation-associated mediator profiles, including IL-1β, IL-10, TNF-α, and TGF-β. Compound 4 also attenuated oxidative stress and inflammatory signaling in U87 cells. Molecular docking studies supported the experimental findings by revealing favorable binding interactions within the active sites of hCA IX and XII, providing a structural basis for their inhibitory activities. Collectively, these findings identify compounds 4, 9, and 30 as promising DTC-based compounds for GBM therapy through the combined modulation of tumor-associated CAs, oxidative stress, inflammatory, and cell death-related pathways.

PMID 42943666
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PubMedMaterials (Basel, Switzerland)2026-10-27

Enhancement of Conversion Efficiency of B(In)GaAs Solar Cells: A Simulation and Numerical Investigation.

Slimi Dalanda D, Saidi Faouzi F, Helali Abdelhamid A, Aissat Abdelkader A et al.

This study focuses on the modeling and optimization of BxGa1-xAs/InyGa1-yAs double-junction photovoltaic cells and GaAs/BxInyGa1-x-yAs quantum well pin cells. It relies on Silvaco Atlas software 2.10.26R to simulate these structures, considering the desired number of QWs or the constant percentage of boron and indium in the III-V-based alloys of BxGa1-xAs, InyGa1-yAs, and BxInyGa1-x-yAs. The BxInyGa1-x-yAs material finds its application in the fabrication of quantum wells incorporated into the intrinsic GaAs-type absorber layer. This absorber layer is positioned between the heavily doped base and the emitting regions, in order to generate a uniform field that allows all photogenerated carriers to escape from the QWs. As part of the modeling, a comparison was made between a 40-layer GaAs/BxInyGa1-x-yAs quantum well solar cell and a standard GaAs solar cell. This analysis revealed an increase in the conversion efficiency of the simulation results from 22.66% to 31.73%. As a result, the integration of quantum wells into the structure of solar cells seems promising to optimize their performance. The optimized BxGa1-xAs/InyGa1-yAs tandem structure demonstrated a short-circuit current density of 17.50 mA/cm2, an open-circuit voltage of 1.99 V, a fill factor of 87.29%, and a conversion efficiency of 30.40% under AM1.5G illumination at 300 K.

PMID 42941012
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PubMedMedicine2026-10-27

The association between inflammation and pubertal development: Insights from NHANES and Mendelian randomization analyses.

Chen Daye D, Qian Lei L, Mei Peng P, Qin Jie J et al.

Our research seeks to elucidate the interplay between inflammation and pubertal development using association and Mendelian randomization (MR) analyses, aiming to uncover the underlying potential causal relationship. Using data from the 2013 to 2016 and 2021 to 2023 National Health and Nutrition Examination Survey cycles, we assessed 3571 children and adolescents. To evaluate associations with pubertal status, logistic regression was performed for a panel of immune-inflammatory biomarkers: platelet-to-lymphocyte ratio, monocyte-to-lymphocyte ratio, neutrophil-to-lymphocyte ratio, systemic inflammation response index, systemic immune-inflammation index, and pan-immune-inflammation value. Bidirectional 2-sample MR analysis was conducted: genetic instruments for exposures were from a published genome-wide protein quantitative trait locus study (91 inflammatory proteins), and outcomes were precocious puberty (PP) summary statistics from the FinnGen consortium R11 database to infer causality. The median age of participants was 10.00 years, with 1838 males (51.47%) and 1733 females (48.53%). In association analysis, neutrophil-to-lymphocyte ratio showed a significant positive association with pubertal status across all models (fully adjusted: odds ratio [OR] = 1.198, 95% confidence interval [CI]: 1.095-1.311, P < .001). Systemic inflammation response index and systemic immune-inflammation index presented significant positive associations only in the fully adjusted model. Forward MR analysis suggested that genetically predicted higher levels of matrix metalloproteinase-10 (OR = 2.213, 95% CI: 1.218-4.024, P = .009) and osteoprotegerin (OR = 2.010, 95% CI: 1.082-3.734, P = .027) were associated with increased risk of PP, while higher tumor necrosis factor-β (TNF-β, OR = 0.678, 95% CI: 0.479-0.959, P = .028) decreased it. Reverse MR showed PP was positively associated with C-X-C motif chemokine ligand 1 (CXCL-1), fibroblast growth factor 5 (FGF-5), fibroblast growth factor 19 (FGF-19), interleukin-7, interleukin-15 receptor subunit alpha, neurturin, and negatively associated with tumor necrosis factor-related activation-induced cytokine. Inflammatory processes may contribute to PP pathogenesis via pathways influencing pubertal timing and progression, providing new insights into pubertal development mechanisms and a scientific basis for future interventions.

PMID 42937763
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